Journal
BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
Volume 17, Issue 1, Pages 78-85Publisher
ELSEVIER SCIENCE INC
DOI: 10.1016/j.bbmt.2010.06.011
Keywords
Acquired bone marrow failure; Alternative donor transplant
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Funding
- Aplastic Anemia and Myelodysplastic Syndrome International Foundation
- France Hemoglobinurie Paroxystique Nocturne
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Information is scarce on outcomes after unrelated cord blood transplantation (UCBT) for patients with severe aplastic anemia (SAA). We retrospectively analyzed 71 patients (median age, 13 years; 28 adults) with SAA (9 with paroxysmal nocturnal hemoglobinuria [PNH]) who received a single-unit (n = 57; 79%) or double-unit UCBT (n = 14; 19%) in 32 centers between 1996 and 2009. A reduced-intensity conditioning regimen was provided in 68% of the patients. The cumulative incidence (Cl) of neutrophil recovery was 51% +/- 6% at day 60, with significantly better engraftment seen in recipients of higher prefreezing total nucleated cell (TNC) dose (>3.9 10(7)/kg; hazard ratio [HR], 1.5; P = .05). The Cl of platelet engraftment at day 180 posttransplantation was 37% +/- 7%, that of grade II-IV acute GVHD was 20% +/- 5%, and that of chronic GVHD at 3 years was 18% +/- 5%. At a median follow-up of 35 months (range, 3-83 months), the estimated probability of 3-year overall survival (OS) was 38% +/- 6%. Significantly improved OS was seen in recipients of >3.9 10(7) TNCs/kg prefreezing (45%, compared with 18% for recipients of <= 3.9 10(7) TNC/kg; HR, 0.4; P = .007). These results highlight the fundamental role of cell dose for both engraftment and OS in patients with SAA undergoing UCBT. Biol Blood Marrow Transplant 17: 78-85 (2011) (C) 2011 American Society for Blood and Marrow Transplantation
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