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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.

Original publication

DOI

10.1182/blood-2013-03-489112

Type

Journal article

Journal

Blood

Publication Date

08/08/2013

Volume

122

Pages

1072 - 1078

Keywords

Adolescent, Adult, Blood Platelets, Bone Marrow Transplantation, Child, Child, Preschool, Disease-Free Survival, Female, Fetal Blood, Graft Survival, Graft vs Host Disease, HLA Antigens, Hemoglobinopathies, Humans, Infant, Male, Multivariate Analysis, Neutrophils, Siblings, Treatment Outcome, Young Adult