Stem cell transplantation in severe congenital neutropenia: an analysis from the European Society for Blood and Marrow Transplantation
Fioredda, Francesca; Iacobelli, Simona; van Biezen, Anja; Gaspar, Bobby; Ancliff, Phil; Donadieu, Jean; Aljurf, Mahmoud; Peters, Christina; Calvillo, Michaela; Matthes-Martin, Susanne; Morreale, Giuseppe; van 't Veer-Tazelaar, Nelleke; de Wreede, Liesbeth; Al Seraihy, Amal; Yesilipek, Akif; Fischer, Alain; Bierings, Marc; Ozturk, Gulyuz; Smith, Owen; Veys, Paul; Ljungman, Per; Peffault de Latour, Régis; Sánchez de Toledo Codina, José; Or, Reuven; Ganser, Arnold; Afanasyev, Boris; Wynn, Robert; Kalwak, Krzysztof; Marsh, Judith; Dufour, Carlo; Severe Aplastic Anemia the Inborn Error, and the Pediatric Disease Working Parties of the European Society for Blood and Bone Marrow Transplantation (EBMT) and Stem Cell Transplant for Immunodeficiencies in Europe (SCETIDE)
(2015) Blood, volume 126, issue 16, pp. 1885 - 1892
(Article)
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
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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.
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Keywords: Clinical Trial, Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't
ISSN: 0006-4971
Publisher: American Society of Hematology
Note: © 2015 by The American Society of Hematology.
(Peer reviewed)