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Recommendations on hematopoietic stem cell transplantation for inherited bone marrow failure syndromes

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Publication date

2015-09

Authors

de Latour, R. Peffault
Peters, C.
Gibsons, B.
Strahm, B.
Lankester, A.
de Heredia, C. D.
Longoni, D.
Fioredda, F.
Locatelli, F.
Yaniv, I.

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DOI

https://doi.org/10.1038/bmt.2015.117

Document Type

Article

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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 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.

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

Citation

de Latour, R P, 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, 'Recommendations on hematopoietic stem cell transplantation for inherited bone marrow failure syndromes', Bone Marrow Transplantation, vol. 50, no. 9, pp. 1168-1172. https://doi.org/10.1038/bmt.2015.117

URI

https://dspace.library.uu.nl/handle/1874/332326