title: Outcomes after Unrelated Umbilical Cord Blood Transplantation for Children with Osteopetrosis.
abstract: Hematopoietic stem cell transplantation (HSCT) is the only curative treatment for most children with osteopetrosis (OP). Timing of HSCT is critical; therefore, umbilical cord blood transplantation (UCBT) is an attractive option. We analyzed outcomes after UCBT in 51 OP children. Median age at UCBT was 6 months. Seventy-seven percent of the cord blood grafts had 0 or 1 HLA disparity with the recipient. Conditioning regimen was myeloablative (mostly busulfan-based in 84% and treosulfan-based in 10%). Antithymocyte globulin was given to 90% of patients. Median number of total nucleated and CD34+cells infused was 14 × 107/kg and 3.4 × 105/kg, respectively. Median follow-up for survivors was 74 months. Cumulative incidence (CI) of neutrophil recovery was 67% with a median time to recovery of 23 days; 33% of patients had graft failure, 81% of engrafted patients had full donor engraftment, and 19% had mixed donor chimerism. Day 100 CI of acute graft-versus-host disease (grades II to IV) was 31% and 6-year CI of chronic graft-versus-host disease was 21%. Mechanical ventilation was required in 28%, and veno-occlusive disease was diagnosed in 16% of cases. Six-year overall survival rate was 46%. Comparative studies with other alternative donors should be performed to evaluate whether UCBT remains a valid alternative for children with OP without an HLA-matched donor.
authors.affiliation: Bone Marrow Transplant Department, Great Ormond Street Children Hospital, London, United Kingdom. Electronic address: robert.chiesa@gosh.nhs.uk.
authors.forename: Robert
authors.initials: R
authors.lastname: Chiesa
authors.affiliation: Eurocord, Hopital Saint Louis and Hopital Saint Antoine, Paris, France.
authors.forename: Annalisa
authors.initials: A
authors.lastname: Ruggeri
authors.affiliation: Eurocord, Hopital Saint Louis and Hopital Saint Antoine, Paris, France.
authors.forename: Annalisa
authors.initials: A
authors.lastname: Paviglianiti
authors.affiliation: Oncoematologia Pediatrica, IRCCS Policlinico San Matteo, Pavia, Italy.
authors.forename: Marco
authors.initials: M
authors.lastname: Zecca
authors.affiliation: Stem Cell Transplant Unit, Hospital Niño Jesús, Madrid, Spain.
authors.forename: Marta
authors.initials: M
authors.lastname: Gónzalez-Vicent
authors.affiliation: Pediatric Hematology, Oncology and Stem Cell Transplantation, Ghent University Hospital, Ghent, Belgium.
authors.forename: Victoria
authors.initials: V
authors.lastname: Bordon
authors.affiliation: Pediatric Hematology Oncology, Schneider Children's Medical Center of Israel, Sackler Faculty of Medicine, Tel Aviv University, Petah Tikva, Israel.
authors.forename: Jerry
authors.initials: J
authors.lastname: Stein
authors.affiliation: Haematology, Birmingham Children's Hospital, Birmingham, United Kingdom.
authors.forename: Sarah
authors.initials: S
authors.lastname: Lawson
authors.affiliation: Section of Hematology, Cliniques Universitaires Saint-Luc, Brussels, Belgium.
authors.forename: Sophie
authors.initials: S
authors.lastname: Dupont
authors.affiliation: Department of Pediatric Hematology/Oncology, G. Gaslini Children's Research Institute, Genova, Italy.
authors.forename: Edoardo
authors.initials: E
authors.lastname: Lanino
authors.affiliation: Serviço de Hematologia, Instituto Portugues de Oncologia, Lisboa, Portugal.
authors.forename: Manuel
authors.initials: M
authors.lastname: Abecasis
authors.affiliation: Department of Pediatric Hematology/Oncology, King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia.
authors.forename: Amal
authors.initials: A
authors.lastname: Al-Seraihy
authors.affiliation: Eurocord, Hopital Saint Louis, Paris, France; Centre Scientifique de Monaco, Principality of Monaco.
authors.forename: Chantal
authors.initials: C
authors.lastname: Kenzey
authors.affiliation: Pediatric Blood and Bone Marrow Transplantation Program, University Medical Center Utrecht, Utrecht, Netherlands.
authors.forename: Marc
authors.initials: M
authors.lastname: Bierings
authors.affiliation: Dipartimento di Onco-Ematologia Pediatrica e Medicina Trasfusionale, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Università di Pavia, Italy.
authors.forename: Franco
authors.initials: F
authors.lastname: Locatelli
authors.affiliation: Eurocord, Hopital Saint Louis, Paris, France; Centre Scientifique de Monaco, Principality of Monaco.
authors.forename: Eliane
authors.initials: E
authors.lastname: Gluckman
authors.affiliation: Department of Pediatrics, University Medical Center Ulm, Ulm, Germany.
authors.forename: Ansgar
authors.initials: A
authors.lastname: Schulz
authors.affiliation: Dept. of Paediatric Immunology, Newcastle General Hospital, Newcastle, United Kingdom.
authors.forename: Andrew
authors.initials: A
authors.lastname: Gennery
authors.affiliation: Pediatric Blood and Marrow Transplant Program, Duke University Medical Center, Durham, North Carolina.
authors.forename: Kristin
authors.initials: K
authors.lastname: Page
authors.affiliation: Churchill Hospital, University of Oxford, NHS Blood and Transplant (NHSBT), Oxford, United Kingdom.
authors.forename: Joanne
authors.initials: J
authors.lastname: Kurtzberg
authors.affiliation: Eurocord, Hopital Saint Louis, Paris, France; Centre Scientifique de Monaco, Principality of Monaco; Churchill Hospital, University of Oxford, NHS Blood and Transplant (NHSBT), Oxford, United Kingdom.
authors.forename: Vanderson
authors.initials: V
authors.lastname: Rocha
mesh_terms: D002648:Child; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods* / Q000401:mortality; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D009504:Neutrophils; D010022:Osteopetrosis / Q000401:mortality / Q000628:therapy*; D020127:Recovery of Function; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000379:methods; D016896:Treatment Outcome; D061349:Unrelated Donors
keywords: Osteopetrosis; Stem cell transplantation; Umbilical cord blood transplantation