Recommendations on hematopoietic stem cell transplantation for inherited bone marrow failure syndromes
de Latour, R. Peffault; Peters, C.; Gibsons, B.; Strahm, B.; Lankester, A.; de Heredia, C. D.; Longoni, D.; Fioredda, F.; Locatelli, F.; Yaniv, I.; Wachowiak, J.; Donadieu, J.; Lawitschka, A.; Bierings, M.; Wlodarski, M.; Corbacioglu, S.; Bonanomi, S.; Samarasinghe, S.; Leblanc, T.; Dufour, C.; Dalle, J-H; Pediat Working Party PDWP; European Grp Blood Marrow
(2015) Bone Marrow Transplantation, volume 50, issue 9, pp. 1168 - 1172
(Article)
Abstract
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
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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.
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Author keywords: SEVERE CONGENITAL NEUTROPENIA, SHWACHMAN-DIAMOND-SYNDROME, SEVERE APLASTIC-ANEMIA, COLONY-STIMULATING FACTOR, ACUTE MYELOID-LEUKEMIA, FANCONI-ANEMIA, DYSKERATOSIS-CONGENITA, EUROPEAN GROUP, MYELODYSPLASTIC SYNDROME, BLOOD
ISSN: 0268-3369
(Publisher version, Peer reviewed)