1. Stem cell transplantation in severe congenital neutropenia: an analysis from the European Society for Blood and Marrow Transplantation.
- Author
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Fioredda F, Iacobelli S, van Biezen A, Gaspar B, Ancliff P, Donadieu J, Aljurf M, Peters C, Calvillo M, Matthes-Martin S, Morreale G, van 't Veer-Tazelaar N, de Wreede L, Al Seraihy A, Yesilipek A, Fischer A, Bierings M, Ozturk G, Smith O, Veys P, Ljungman P, Peffault de Latour R, Sánchez de Toledo Codina J, Or R, Ganser A, Afanasyev B, Wynn R, Kalwak K, Marsh J, and Dufour C
- Subjects
- Acute Disease, Adolescent, Adult, Allografts, Child, Child, Preschool, Europe epidemiology, Female, Histocompatibility Testing, Humans, Incidence, Male, Middle East, Retrospective Studies, Societies, Medical, Cyclosporine administration & dosage, Graft Rejection epidemiology, Graft Rejection prevention & control, Graft vs Host Disease epidemiology, Graft vs Host Disease prevention & control, Hematopoietic Stem Cell Transplantation, Methotrexate administration & dosage, Neutropenia congenital, Neutropenia epidemiology, Neutropenia therapy, Unrelated Donors
- Abstract
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment of severe congenital neutropenia (SCN), but data on outcome are scarce. We report on the outcome of 136 SCN patients who underwent HSCT between 1990 and 2012 in European and Middle East centers. The 3-year overall survival (OS) was 82%, and transplant-related mortality (TRM) was 17%. In multivariate analysis, transplants performed under the age of 10 years, in recent years, and from HLA-matched related or unrelated donors were associated with a significantly better OS. Frequency of graft failure was 10%. Cumulative incidence (day +90) of acute graft-versus-host disease (GVHD) grade 2-4 was 21%. In multivariate analysis, HLA-matched related donor and prophylaxis with cyclosporine A and methotrexate were associated with lower occurrence of acute GVHD. Cumulative incidence (1 year) of chronic GVHD was 20%. No secondary malignancies occurred after a median follow-up of 4.6 years. These data show that the outcome of HSCT for SCN from HLA-matched donors, performed in recent years, in patients younger than 10 years is acceptable. Nevertheless, given the TRM, a careful selection of HSCT candidates should be undertaken., (© 2015 by The American Society of Hematology.)
- Published
- 2015
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