TY - JOUR
T1 - Recommendations on hematopoietic stem cell transplantation for inherited bone marrow failure syndromes
AU - de Latour, R. Peffault
AU - Peters, C.
AU - Gibsons, B.
AU - Strahm, B.
AU - Lankester, A.
AU - de Heredia, C. D.
AU - Longoni, D.
AU - Fioredda, F.
AU - Locatelli, F.
AU - Yaniv, I.
AU - Wachowiak, J.
AU - Donadieu, J.
AU - Lawitschka, A.
AU - Bierings, M.
AU - Wlodarski, M.
AU - Corbacioglu, S.
AU - Bonanomi, S.
AU - Samarasinghe, S.
AU - Leblanc, T.
AU - Dufour, C.
AU - Dalle, J-H
PY - 2015/9
Y1 - 2015/9
N2 - Allogeneic hematopoietic stem cell transplantation (HSCT) offers the potential to cure patients with an inherited bone marrow failure syndrome (IBMFS). However, the procedure involves the risk of treatment-related mortality and may be associated with significant early and late morbidity. For these reasons, the benefits should be carefully weighed against the risks. IBMFS are rare, whereas case reports and small series in the literature illustrate highly heterogeneous practices in terms of indications for HSCT, timing, stem cell source and conditioning regimens. A consensus meeting was therefore held in Vienna in September 2012 on behalf of the European Group for Blood and Marrow Transplantation to discuss HSCT in the setting of IBMFS. This report summarizes the recommendations from this expert panel, including indications for HSCT, timing, stem cell source and conditioning regimen.
AB - Allogeneic hematopoietic stem cell transplantation (HSCT) offers the potential to cure patients with an inherited bone marrow failure syndrome (IBMFS). However, the procedure involves the risk of treatment-related mortality and may be associated with significant early and late morbidity. For these reasons, the benefits should be carefully weighed against the risks. IBMFS are rare, whereas case reports and small series in the literature illustrate highly heterogeneous practices in terms of indications for HSCT, timing, stem cell source and conditioning regimens. A consensus meeting was therefore held in Vienna in September 2012 on behalf of the European Group for Blood and Marrow Transplantation to discuss HSCT in the setting of IBMFS. This report summarizes the recommendations from this expert panel, including indications for HSCT, timing, stem cell source and conditioning regimen.
KW - SEVERE CONGENITAL NEUTROPENIA
KW - SHWACHMAN-DIAMOND-SYNDROME
KW - SEVERE APLASTIC-ANEMIA
KW - COLONY-STIMULATING FACTOR
KW - ACUTE MYELOID-LEUKEMIA
KW - FANCONI-ANEMIA
KW - DYSKERATOSIS-CONGENITA
KW - EUROPEAN GROUP
KW - MYELODYSPLASTIC SYNDROME
KW - BLOOD
U2 - 10.1038/bmt.2015.117
DO - 10.1038/bmt.2015.117
M3 - Article
SN - 0268-3369
VL - 50
SP - 1168
EP - 1172
JO - Bone Marrow Transplantation
JF - Bone Marrow Transplantation
IS - 9
ER -