title: Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling.
abstract: We analyzed the outcomes of 485 patients with thalassemia major (TM) or sickle cell disease (SCD) receiving HLA-identical sibling cord blood transplantation (CBT, n = 96) or bone marrow transplantation (BMT, n = 389). Compared with patients given BMT, CBT recipients were significantly younger (median age 6 vs 8 years, P = .02), and were treated more recently (median year 2001 vs 1999, P < .01). A higher proportion of patients with TM belonging to classes II-III of the Pesaro classification received BMT (44%) compared with CBT (39%, P < .01). In comparison with patients receiving BMT (n = 259, TM; n = 130, SCD), those given CBT (n = 66, TM; n = 30, SCD) had slower neutrophil recovery, less acute graft-versus-host disease (GVHD) and none had extensive chronic GVHD. With a median follow-up of 70 months, the 6-year overall survival was 95% and 97% after BMT and CBT, respectively (P = .92). The 6-year disease-free survival (DFS) was 86% and 80% in TM patients after BMT and CBT, respectively, whereas DFS in SCD patients was 92% and 90%, respectively. The cell dose infused did not influence outcome of patients given CBT. In multivariate analysis, DFS did not differ between CBT and BMT recipients. Patients with TM or SCD have excellent outcomes after both HLA-identical sibling CBT and BMT.
authors.affiliation: Department of Pediatric Hematology-Oncology, Istituto di Ricovero e Cura a Carattere Scientifico, Ospedale Pediatrico Bambino Gesù, Piazzale Sant'Onofrio 4, Rome, Italy. franco.locatelli@opbg.net
authors.forename: Franco
authors.initials: F
authors.lastname: Locatelli
authors.forename: Nabil
authors.initials: N
authors.lastname: Kabbara
authors.forename: Annalisa
authors.initials: A
authors.lastname: Ruggeri
authors.forename: Ardeshir
authors.initials: A
authors.lastname: Ghavamzadeh
authors.forename: Irene
authors.initials: I
authors.lastname: Roberts
authors.forename: Chi Kong
authors.initials: CK
authors.lastname: Li
authors.forename: Françoise
authors.initials: F
authors.lastname: Bernaudin
authors.forename: Christiane
authors.initials: C
authors.lastname: Vermylen
authors.forename: Jean-Hugues
authors.initials: JH
authors.lastname: Dalle
authors.forename: Jerry
authors.initials: J
authors.lastname: Stein
authors.forename: Robert
authors.initials: R
authors.lastname: Wynn
authors.forename: Catherine
authors.initials: C
authors.lastname: Cordonnier
authors.forename: Fernando
authors.initials: F
authors.lastname: Pinto
authors.forename: Emanuele
authors.initials: E
authors.lastname: Angelucci
authors.forename: Gérard
authors.initials: G
authors.lastname: Socié
authors.forename: Eliane
authors.initials: E
authors.lastname: Gluckman
authors.forename: Mark C
authors.initials: MC
authors.lastname: Walters
authors.forename: Vanderson
authors.initials: V
authors.lastname: Rocha
mesh_terms: D000293:Adolescent; D000328:Adult; D001792:Blood Platelets / Q000166:cytology; D016026:Bone Marrow Transplantation / Q000379:methods*; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D005312:Fetal Blood / Q000637:transplantation*; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000628:therapy; D006680:HLA Antigens / Q000276:immunology*; D006453:Hemoglobinopathies / Q000276:immunology* / Q000628:therapy*; D006801:Humans; D007223:Infant; D008297:Male; D015999:Multivariate Analysis; D009504:Neutrophils / Q000166:cytology / Q000378:metabolism; D035781:Siblings; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D006680:HLA Antigens