1. Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling
- Author
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Jerry Stein, Françoise Bernaudin, Fernando Pinto, Ardeshir Ghavamzadeh, Catherine Cordonnier, Emanuele Angelucci, Nabil Kabbara, Annalisa Ruggeri, Chi Kong Li, Irene Roberts, Gérard Socié, Christiane Vermylen, Jean-Hugues Dalle, Robert Wynn, Eliane Gluckman, Vanderson Rocha, Mark C. Walters, Franco Locatelli, Eurocord, UCL - (SLuc) Service d'hématologie et d'oncologie pédiatrique, UCL - SSS/IREC/PEDI - Pôle de Pédiatrie, and UCL - (SLuc) Service d'hématologie et d'oncologie pédiatrique
- Subjects
Adult ,Blood Platelets ,Male ,medicine.medical_specialty ,Adolescent ,Neutrophils ,Thalassemia ,Immunology ,Graft vs Host Disease ,Human leukocyte antigen ,behavioral disciplines and activities ,Biochemistry ,Gastroenterology ,Disease-Free Survival ,Young Adult ,HLA Antigens ,hemic and lymphatic diseases ,Internal medicine ,medicine ,Humans ,Sibling ,Young adult ,Child ,Bone Marrow Transplantation ,business.industry ,Umbilical Cord Blood Transplantation ,Siblings ,Graft Survival ,Infant ,Cell Biology ,Hematology ,medicine.disease ,Fetal Blood ,Surgery ,Transplantation ,Hemoglobinopathies ,surgical procedures, operative ,Graft-versus-host disease ,Treatment Outcome ,N/A ,Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA ,Cord blood ,Child, Preschool ,Multivariate Analysis ,Female ,business - 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.
- Published
- 2013