TY - JOUR
T1 - Stem cell transplantation in severe congenital neutropenia
T2 - an analysis from the European Society for Blood and Marrow Transplantation
AU - Fioredda, Francesca
AU - Iacobelli, Simona
AU - van Biezen, Anja
AU - Gaspar, Bobby
AU - Ancliff, Phil
AU - Donadieu, Jean
AU - Aljurf, Mahmoud
AU - Peters, Christina
AU - Calvillo, Michaela
AU - Matthes-Martin, Susanne
AU - Morreale, Giuseppe
AU - van 't Veer-Tazelaar, Nelleke
AU - de Wreede, Liesbeth
AU - Al Seraihy, Amal
AU - Yesilipek, Akif
AU - Fischer, Alain
AU - Bierings, Marc
AU - Ozturk, Gulyuz
AU - Smith, Owen
AU - Veys, Paul
AU - Ljungman, Per
AU - Peffault de Latour, Régis
AU - Sánchez de Toledo Codina, José
AU - Or, Reuven
AU - Ganser, Arnold
AU - Afanasyev, Boris
AU - Wynn, Robert
AU - Kalwak, Krzysztof
AU - Marsh, Judith
AU - Dufour, Carlo
N1 - © 2015 by The American Society of Hematology.
PY - 2015
Y1 - 2015
N2 - 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.
AB - 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.
U2 - 10.1182/blood-2015-02-628859
DO - 10.1182/blood-2015-02-628859
M3 - Article
C2 - 26185129
SN - 0006-4971
VL - 126
SP - 1885
EP - 1892
JO - Blood
JF - Blood
IS - 16
ER -