Stem cell transplantation in severe congenital neutropenia: An analysis from the European Society for Blood and Marrow Transplantation

Francesca Fioredda, Simona Iacobelli, Anja Van Biezen, Bobby Gaspar, Phil Ancliff, Jean Donadieu, Mahmoud Aljurf, Christina Peters, Michaela Calvillo, Susanne Matthes-Martin, Giuseppe Morreale, Nelleke Van't Veer-Tazelaar, Liesbeth De Wreede, Amal Al Seraihy, Akif Yesilipek, Alain Fischer, Marc Bierings, Gulyuz Ozturk, Owen Smith, Paul VeysPer Ljungman, Régis Peffault De Latour, José Sánchez De Toledo Codina, Reuven Or, Arnold Ganser, Boris Afanasyev, Robert Wynn, Krzysztof Kalwak, Judith Marsh, Carlo Dufour

Research output: Contribution to journalArticle

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment of severe congenital neutropenia (SCN), but data on outcome are scarce.Wereport 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 190) 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.

Original languageEnglish
Pages (from-to)1885-1892
Number of pages8
JournalBlood
Volume126
Issue number16
DOIs
Publication statusPublished - Oct 15 2015

ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology
  • Medicine(all)

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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., ... Dufour, C. (2015). Stem cell transplantation in severe congenital neutropenia: An analysis from the European Society for Blood and Marrow Transplantation. Blood, 126(16), 1885-1892. https://doi.org/10.1182/blood-2015-02-628859