Abstract |
We examined the efficacy of unrelated cord blood (CB) transplantation in children with thalassemia (n = 35) and sickle cell disease (n = 16), using data reported to 3 registries. Donor-recipient pairs were matched at HLA-A and -B ( antigen level) and DRB1 (allele level) in 7 or HLA mismatched at 1 (n = 18), 2 (n = 25), or 3 loci (n = 1). Transplant conditioning was myeloablative (n = 39) or reduced intensity (n = 12). Neutrophil recovery with donor chimerism was documented in 24 patients; 11 patients developed grade II-IV acute graft-versus-host disease (aGVHD) and 10 patients, chronic GVHD (cGVHD). Overall survival (OS) and disease-free survival (DFS) were 62% and 21% for thalassemia and 94% and 50% for sickle cell disease (SCD), respectively. In multivariate analysis, engraftment rate (hazard ratio [HR] 2.2, P = .05) and DFS (HR 0.4, P = .01) were higher with cell dose >5 × 10(7)/kg. The 2-year probability of DFS was 45% in patients who received grafts with cell dose >5 × 10(7)/kg and 13% with lower cell dose. Primary graft failure was the predominant cause of treatment failure occurring in 20 patients with thalassemia and 7 patients with SCD. Primary graft failure was fatal in 5 patients with thalassemia. These results suggest that only CB units containing an expected infused cell dose >5 × 10(7)/kg should be considered for transplantation for hemoglobinopathy.
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Authors | Annalisa Ruggeri, Mary Eapen, Andromachi Scaravadou, Mitchell S Cairo, Monica Bhatia, Joanne Kurtzberg, John R Wingard, Anders Fasth, Luca Lo Nigro, Mouhab Ayas, Duncan Purtill, Karim Boudjedir, Wagnara Chaves, Mark C Walters, John Wagner, Eliane Gluckman, Vanderson Rocha, Eurocord Registry, Center for International Blood and Marrow Transplant Research, New York Blood Center |
Journal | Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
(Biol Blood Marrow Transplant)
Vol. 17
Issue 9
Pg. 1375-82
(Sep 2011)
ISSN: 1523-6536 [Electronic] United States |
PMID | 21277376
(Publication Type: Journal Article, Research Support, N.I.H., Extramural)
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Copyright | Copyright © 2011 American Society for Blood and Marrow Transplantation. All rights reserved. |
Topics |
- Anemia, Sickle Cell
(mortality, therapy)
- Cell Count
- Child
- Cord Blood Stem Cell Transplantation
(adverse effects, methods, statistics & numerical data)
- Female
- Graft Survival
- Graft vs Host Disease
- Histocompatibility Testing
- Humans
- Male
- Neutrophils
- Registries
- Survival Analysis
- Thalassemia
(mortality, therapy)
- Transplantation Chimera
- Transplantation Conditioning
(methods)
- Treatment Outcome
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