title: Primary Immune Deficiency Treatment Consortium (PIDTC) report.
abstract: The Primary Immune Deficiency Treatment Consortium (PIDTC) is a network of 33 centers in North America that study the treatment of rare and severe primary immunodeficiency diseases. Current protocols address the natural history of patients treated for severe combined immunodeficiency (SCID), Wiskott-Aldrich syndrome, and chronic granulomatous disease through retrospective, prospective, and cross-sectional studies. The PIDTC additionally seeks to encourage training of junior investigators, establish partnerships with European and other International colleagues, work with patient advocacy groups to promote community awareness, and conduct pilot demonstration projects. Future goals include the conduct of prospective treatment studies to determine optimal therapies for primary immunodeficiency diseases. To date, the PIDTC has funded 2 pilot projects: newborn screening for SCID in Navajo Native Americans and B-cell reconstitution in patients with SCID after hematopoietic stem cell transplantation. Ten junior investigators have received grant awards. The PIDTC Annual Scientific Workshop has brought together consortium members, outside speakers, patient advocacy groups, and young investigators and trainees to report progress of the protocols and discuss common interests and goals, including new scientific developments and future directions of clinical research. Here we report the progress of the PIDTC to date, highlights of the first 2 PIDTC workshops, and consideration of future consortium objectives.
authors.affiliation: Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md. Electronic address: LGriffith@niaid.nih.gov.
authors.forename: Linda M
authors.initials: LM
authors.lastname: Griffith
authors.affiliation: Division of Allergy/Immunology and Blood and Marrow Transplantation, Department of Pediatrics and UCSF Benioff Children's Hospital, University of California San Francisco, San Francisco, Calif.
authors.forename: Morton J
authors.initials: MJ
authors.lastname: Cowan
authors.affiliation: Division of Immunology, the Manton Center for Orphan Disease Research, Children's Hospital, and Harvard Stem Cell Institute, Harvard Medical School, Boston, Mass.
authors.forename: Luigi D
authors.initials: LD
authors.lastname: Notarangelo
authors.affiliation: Departments of Microbiology, Immunology and Molecular Genetics, University of California Los Angeles, Los Angeles, Calif.
authors.forename: Donald B
authors.initials: DB
authors.lastname: Kohn
authors.affiliation: Division of Allergy/Immunology and Blood and Marrow Transplantation, Department of Pediatrics and UCSF Benioff Children's Hospital, University of California San Francisco, San Francisco, Calif; Institute for Human Genetics, University of California San Francisco, San Francisco, Calif.
authors.forename: Jennifer M
authors.initials: JM
authors.lastname: Puck
authors.affiliation: Pediatric Hematology/Oncology, Children's Hospital, Harvard Medical School, Boston, Mass.
authors.forename: Sung-Yun
authors.initials: SY
authors.lastname: Pai
authors.affiliation: Immune Deficiency Foundation, Towson, Md.
authors.forename: Barbara
authors.initials: B
authors.lastname: Ballard
authors.affiliation: Developmental and Behavioral Pediatrics, Lurie Children's Hospital of Chicago, Northwestern Feinberg School of Medicine, Chicago, Ill.
authors.forename: Sarah C
authors.initials: SC
authors.lastname: Bauer
authors.affiliation: Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
authors.forename: Jack J H
authors.initials: JJ
authors.lastname: Bleesing
authors.affiliation: Immune Deficiency Foundation, Towson, Md.
authors.forename: Marcia
authors.initials: M
authors.lastname: Boyle
authors.affiliation: Newborn Screening Translational Research Network, American College of Medical Genetics and Genomics, Bethesda, Md.
authors.forename: Amy
authors.initials: A
authors.lastname: Brower
authors.affiliation: Pediatric Allergy and Immunology, Duke University School of Medicine, Durham, NC.
authors.forename: Rebecca H
authors.initials: RH
authors.lastname: Buckley
authors.affiliation: Molecular Immunology, Erasmus Medical Center, Rotterdam, The Netherlands.
authors.forename: Mirjam
authors.initials: M
authors.lastname: van der Burg
authors.affiliation: Pediatric Hematology/Oncology, Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, Wash.
authors.forename: Lauri M
authors.initials: LM
authors.lastname: Burroughs
authors.affiliation: Genetics & Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Md.
authors.forename: Fabio
authors.initials: F
authors.lastname: Candotti
authors.affiliation: Pediatric Immunology and Infectious Diseases and Pediatric Bone Marrow Transplant, Newcastle General Hospital, Newcastle upon Tyne, United Kingdom.
authors.forename: Andrew J
authors.initials: AJ
authors.lastname: Cant
authors.affiliation: Pediatric Allergy/Immunology, Children's Hospital, Harvard Medical School, Boston, Mass.
authors.forename: Talal
authors.initials: T
authors.lastname: Chatila
authors.affiliation: Pediatric Clinical Immunology, Mount Sinai School of Medicine, New York, NY.
authors.forename: Charlotte
authors.initials: C
authors.lastname: Cunningham-Rundles
authors.affiliation: Pediatric Hematology/Oncology, Washington University School of Medicine, St Louis, Mo.
authors.forename: Mary C
authors.initials: MC
authors.lastname: Dinauer
authors.affiliation: Division of Allergy/Immunology and Blood and Marrow Transplantation, Department of Pediatrics and UCSF Benioff Children's Hospital, University of California San Francisco, San Francisco, Calif.
authors.forename: Christopher C
authors.initials: CC
authors.lastname: Dvorak
authors.affiliation: Pediatric Clinical Immunology, Division of Hematology/Oncology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
authors.forename: Alexandra H
authors.initials: AH
authors.lastname: Filipovich
authors.affiliation: Department of Laboratory Medicine, Clinical Center, National Institutes of Health, Bethesda, Md.
authors.forename: Thomas A
authors.initials: TA
authors.lastname: Fleisher
authors.affiliation: Pediatric Immunology, Center for Immunodeficiency, Institute of Child Health, Great Ormond Street Hospital, University College London, London, United Kingdom.
authors.forename: Hubert
authors.initials: H
authors.lastname: Bobby Gaspar
authors.affiliation: Pediatric Immunology and Blood and Marrow Transplantation, Children's Hospital Zurich, University of Zurich, Zurich, Switzerland.
authors.forename: Tayfun
authors.initials: T
authors.lastname: Gungor
authors.affiliation: Pediatric Immunology, Mother and Child Ste-Justine Hospital, Montreal, Quebec, Canada.
authors.forename: Elie
authors.initials: E
authors.lastname: Haddad
authors.affiliation: Immune Deficiency Foundation, Towson, Md.
authors.forename: Emily
authors.initials: E
authors.lastname: Hovermale
authors.affiliation: Pediatric Allergy/Immunology, Mount Sinai Medical Center, New York, NY.
authors.forename: Faith
authors.initials: F
authors.lastname: Huang
authors.affiliation: Chronic Granulomatous Disease Association, San Marino, Calif.
authors.forename: Alan
authors.initials: A
authors.lastname: Hurley
authors.affiliation: Chronic Granulomatous Disease Association, San Marino, Calif.
authors.forename: Mary
authors.initials: M
authors.lastname: Hurley
authors.affiliation: Wiskott-Aldrich Foundation, Smyrna, Ga.
authors.forename: Sumathi
authors.initials: S
authors.lastname: Iyengar
authors.affiliation: Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md.
authors.forename: Elizabeth M
authors.initials: EM
authors.lastname: Kang
authors.affiliation: Center for International Blood and Marrow Transplant Research and Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wis.
authors.forename: Brent R
authors.initials: BR
authors.lastname: Logan
authors.affiliation: Department of Clinical Pharmacy, School of Pharmacy, University of California, San Francisco, Calif.
authors.forename: Janel R
authors.initials: JR
authors.lastname: Long-Boyle
authors.affiliation: Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md.
authors.forename: Harry L
authors.initials: HL
authors.lastname: Malech
authors.affiliation: Pediatric Allergy/Immunology, Lucile Packard Children's Hospital, Stanford University Medical Center, Stanford, Calif.
authors.forename: Sean A
authors.initials: SA
authors.lastname: McGhee
authors.affiliation: Jeffrey Modell Foundation, New York, NY.
authors.forename: Fred
authors.initials: F
authors.lastname: Modell
authors.affiliation: Jeffrey Modell Foundation, New York, NY.
authors.forename: Vicki
authors.initials: V
authors.lastname: Modell
authors.affiliation: Center for Immunity and Immunotherapy, Seattle Children's Hospital Research Institute, University of Washington School of Medicine, Seattle, Wash.
authors.forename: Hans D
authors.initials: HD
authors.lastname: Ochs
authors.affiliation: Pediatrics and Immunology, Memorial Sloan Kettering Cancer Center, New York, NY.
authors.forename: Richard J
authors.initials: RJ
authors.lastname: O'Reilly
authors.affiliation: Division of Research Immunology/B.M.T., Children's Hospital Los Angeles, Los Angeles, Calif.
authors.forename: Robertson
authors.initials: R
authors.lastname: Parkman
authors.affiliation: Pediatric Immunology, Seattle Children's Research Institute, University of Washington School of Medicine, Seattle, Wash.
authors.forename: David J
authors.initials: DJ
authors.lastname: Rawlings
authors.affiliation: Pediatric Allergy and Clinical Immunology, Children's Hospital of Wisconsin, Medical College of Wisconsin, Milwaukee, Wis.
authors.forename: John M
authors.initials: JM
authors.lastname: Routes
authors.affiliation: Pediatric Allergy & Immunology, Texas Children's Hospital, Baylor College of Medicine, Houston, Tex.
authors.forename: William T
authors.initials: WT
authors.lastname: Shearer
authors.affiliation: Pediatric Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, NY.
authors.forename: Trudy N
authors.initials: TN
authors.lastname: Small
authors.affiliation: SCID Angels for Life Foundation, Lakeland, Fla.
authors.forename: Heather
authors.initials: H
authors.lastname: Smith
authors.affiliation: Pediatric Immunology, Children's Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pa.
authors.forename: Kathleen E
authors.initials: KE
authors.lastname: Sullivan
authors.affiliation: Bone Marrow Transplantation and Cellular Therapies, Children's Hospital of Pittsburgh, University of Pittsburgh Medical Center, Pittsburgh, Pa.
authors.forename: Paul
authors.initials: P
authors.lastname: Szabolcs
authors.affiliation: Pediatric Immunology, Center for Immunodeficiency, Institute of Child Health, Great Ormond Street Hospital, University College London, London, United Kingdom.
authors.forename: Adrian
authors.initials: A
authors.lastname: Thrasher
authors.affiliation: Pediatric Rheumatology, Seattle Children's Research Institute, University of Washington School of Medicine, Seattle, Wash.
authors.forename: Troy R
authors.initials: TR
authors.lastname: Torgerson
authors.affiliation: Blood and Marrow Transplantation, Institute of Child Health, Great Ormond Street Hospital, London, United Kingdom.
authors.forename: Paul
authors.initials: P
authors.lastname: Veys
authors.affiliation: Pediatric Stem Cell Transplantation and Hematology/Oncology, Lucile Packard Children's Hospital, Stanford University School of Medicine, Stanford, Calif.
authors.forename: Kenneth
authors.initials: K
authors.lastname: Weinberg
authors.affiliation: Department of Immunology, University of Toronto, Toronto, Ontario, Canada.
authors.forename: Juan Carlos
authors.initials: JC
authors.lastname: Zuniga-Pflucker
mesh_terms: D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000175:diagnosis / Q000628:therapy; D007231:Infant, Newborn; D015997:Neonatal Screening; D010865:Pilot Projects; D012958:Societies, Scientific
keywords: ADA; Adenosine deaminase; Allogeneic hematopoietic cell transplantation; CGD; CIBMTR; Center for International Blood and Marrow Transplant Research; Chronic granulomatous disease; EBMT; ESID; European Group for Blood and Marrow Transplantation; European Society for Immunodeficiencies; GT; GVHD; Gene therapy; Graft-versus-host disease; HCT; Hematopoietic cell transplantation; IEWP; Inborn Errors Working Party; MAC; MUD; Matched unrelated donor; Myeloablative conditioning; NBS; NIAID; NIH; NK; National Institute of Allergy and Infectious Diseases; National Institutes of Health; Natural killer; Newborn screening for SCID; PID; PIDTC; Primary Immune Deficiency Treatment Consortium; Primary immunodeficiency; RIC; Reduced-intensity conditioning; SCETIDE; SCID; Severe combined immunodeficiency; Stem Cell Transplantation for Immunodeficiencies in Europe; USIDNET; United States Immunodeficiency Network; WAS; Wiskott-Aldrich syndrome; clinical trial; gene therapy; primary immunodeficiency