title: A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A.
abstract: BACKGROUND
  The development of neutralizing anti-factor VIII alloantibodies (inhibitors) in patients with severe hemophilia A may depend on the concentrate used for replacement therapy.
  
  
  METHODS
  We conducted a randomized trial to assess the incidence of factor VIII inhibitors among patients treated with plasma-derived factor VIII containing von Willebrand factor or recombinant factor VIII. Patients who met the eligibility criteria (male sex, age <6 years, severe hemophilia A, and no previous treatment with any factor VIII concentrate or only minimal treatment with blood components) were included from 42 sites.
  
  
  RESULTS
  Of 303 patients screened, 264 underwent randomization and 251 were analyzed. Inhibitors developed in 76 patients, 50 of whom had high-titer inhibitors (≥5 Bethesda units). Inhibitors developed in 29 of the 125 patients treated with plasma-derived factor VIII (20 patients had high-titer inhibitors) and in 47 of the 126 patients treated with recombinant factor VIII (30 patients had high-titer inhibitors). The cumulative incidence of all inhibitors was 26.8% (95% confidence interval [CI], 18.4 to 35.2) with plasma-derived factor VIII and 44.5% (95% CI, 34.7 to 54.3) with recombinant factor VIII; the cumulative incidence of high-titer inhibitors was 18.6% (95% CI, 11.2 to 26.0) and 28.4% (95% CI, 19.6 to 37.2), respectively. In Cox regression models for the primary end point of all inhibitors, recombinant factor VIII was associated with an 87% higher incidence than plasma-derived factor VIII (hazard ratio, 1.87; 95% CI, 1.17 to 2.96). This association did not change in multivariable analysis. For high-titer inhibitors, the hazard ratio was 1.69 (95% CI, 0.96 to 2.98). When the analysis was restricted to recombinant factor VIII products other than second-generation full-length recombinant factor VIII, effect estimates remained similar for all inhibitors (hazard ratio, 1.98; 95% CI, 0.99 to 3.97) and high-titer inhibitors (hazard ratio, 2.59; 95% CI, 1.11 to 6.00).
  
  
  CONCLUSIONS
  Patients treated with plasma-derived factor VIII containing von Willebrand factor had a lower incidence of inhibitors than those treated with recombinant factor VIII. (Funded by the Angelo Bianchi Bonomi Foundation and others; ClinicalTrials.gov number, NCT01064284; EudraCT number, 2009-011186-88.).
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Flora
authors.initials: F
authors.lastname: Peyvandi
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Pier M
authors.initials: PM
authors.lastname: Mannucci
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Isabella
authors.initials: I
authors.lastname: Garagiola
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Amal
authors.initials: A
authors.lastname: El-Beshlawy
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Mohsen
authors.initials: M
authors.lastname: Elalfy
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Vijay
authors.initials: V
authors.lastname: Ramanan
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Peyman
authors.initials: P
authors.lastname: Eshghi
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Suresh
authors.initials: S
authors.lastname: Hanagavadi
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Ramabadran
authors.initials: R
authors.lastname: Varadarajan
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Mehran
authors.initials: M
authors.lastname: Karimi
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Mamta V
authors.initials: MV
authors.lastname: Manglani
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Cecil
authors.initials: C
authors.lastname: Ross
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Guy
authors.initials: G
authors.lastname: Young
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Tulika
authors.initials: T
authors.lastname: Seth
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Shashikant
authors.initials: S
authors.lastname: Apte
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Dinesh M
authors.initials: DM
authors.lastname: Nayak
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Elena
authors.initials: E
authors.lastname: Santagostino
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Maria Elisa
authors.initials: ME
authors.lastname: Mancuso
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Adriana C
authors.initials: AC
authors.lastname: Sandoval Gonzalez
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Johnny N
authors.initials: JN
authors.lastname: Mahlangu
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Santiago
authors.initials: S
authors.lastname: Bonanad Boix
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Monica
authors.initials: M
authors.lastname: Cerqueira
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Nadia P
authors.initials: NP
authors.lastname: Ewing
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Christoph
authors.initials: C
authors.lastname: Male
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Tarek
authors.initials: T
authors.lastname: Owaidah
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Veronica
authors.initials: V
authors.lastname: Soto Arellano
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Nathan L
authors.initials: NL
authors.lastname: Kobrinsky
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Suvankar
authors.initials: S
authors.lastname: Majumdar
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Rosario
authors.initials: R
authors.lastname: Perez Garrido
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Anupam
authors.initials: A
authors.lastname: Sachdeva
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Mindy
authors.initials: M
authors.lastname: Simpson
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Mathew
authors.initials: M
authors.lastname: Thomas
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Ezio
authors.initials: E
authors.lastname: Zanon
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Bulent
authors.initials: B
authors.lastname: Antmen
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Kaan
authors.initials: K
authors.lastname: Kavakli
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Marilyn J
authors.initials: MJ
authors.lastname: Manco-Johnson
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Monica
authors.initials: M
authors.lastname: Martinez
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Esperanza
authors.initials: E
authors.lastname: Marzouka
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Maria G
authors.initials: MG
authors.lastname: Mazzucconi
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Daniela
authors.initials: D
authors.lastname: Neme
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Angeles
authors.initials: A
authors.lastname: Palomo Bravo
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Rogelio
authors.initials: R
authors.lastname: Paredes Aguilera
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Alessandra
authors.initials: A
authors.lastname: Prezotti
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Klaus
authors.initials: K
authors.lastname: Schmitt
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Brian M
authors.initials: BM
authors.lastname: Wicklund
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Bulent
authors.initials: B
authors.lastname: Zulfikar
authors.affiliation: From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico (F.P., P.M.M., E.S., M.E.M.), and Department of Pathophysiology and Transplantation, Università degli Studi di Milano (F.P., P.M.M., I.G.), Milan, Clinica Medica II, Azienda Ospedaliera di Padova, Centro Emofilia, Padua (E.Z.), and Ematologia, Unità Operativa Diagnostica Speciale e Terapia delle Malattie dell'Emostasi e della Trombosi, Università Sapienza, Policlinico Umberto I, Rome (M.G.M.) - all in Italy; the Pediatric Hematology Department, Cairo University Pediatric Hospital (A.E.-B.), and Department of Pediatrics, Faculty of Medicine, Ain Shams University (M.E.), Cairo; Jehangir Clinical Development Center, Department of Hematology, Jehangir Hospital Premises (V.R.), and Sahyadri Speciality Hospital (S.A.), Pune, Jagadguru Jayadeva Murugarajendra Medical College, Davangere (S.H.), Center for Blood Disorders, Chennai (R.V.), Lokmanya Tilak Municipal Medical College and General Hospital, Mumbai (M.V.M.); St. John's Medical College Hospital, Bangalore (C.R.), All India Institute of Medical Sciences, Department of Hematology (T.S.), and Pediatric Hematology Oncology and Bone Marrow Transplantation, Institute for Child Health, Sir Ganga Ram Hospital (A.S.), New Delhi, Melaka-Manipal Medical College, Manipal University, Manipal (D.M.N.), and Kerala Institute of Medical Science, Trivandrum (M.T.) - all in India; the Congenital Pediatric Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran (P.E.), and Hematology Research Center, Shiraz University of Medical Sciences, Shiraz (M.K.) - both in Iran; Children's Hospital Los Angeles, Los Angeles (G.Y.), and City of Hope National Medical Center, Duarte (N.P.E.) - both in California; Hospital de Especialidades Unidad Médica de Alta Especialidad, Instituto Mexicano del Seguro Social, Monterrey (A.C.S.G.), an
authors.forename: Frits R
authors.initials: FR
authors.lastname: Rosendaal
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D057134:Antibodies, Neutralizing / Q000097:blood*; D002648:Child; D002675:Child, Preschool; D004305:Dose-Response Relationship, Drug; D004359:Drug Therapy, Combination; D005169:Factor VIII / Q000037:antagonists & inhibitors / Q000276:immunology* / Q000627:therapeutic use; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy* / Q000276:immunology; D006470:Hemorrhage / Q000209:etiology; D006801:Humans; D015994:Incidence; D007223:Infant; D007279:Injections, Subcutaneous / Q000009:adverse effects; D007518:Isoantibodies / Q000032:analysis*; D008297:Male; D008875:Middle Aged; D016016:Proportional Hazards Models; D055815:Young Adult; D014841:von Willebrand Factor / Q000627:therapeutic use*
chemical_list: D057134:Antibodies, Neutralizing; D007518:Isoantibodies; D014841:von Willebrand Factor; C427184:recombinant factor VIII SQ; D005169:Factor VIII