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

de Latour, R. Peffault and Peters, C. and Gibsons, B. and Strahm, B. and Lankester, A. and de Heredia, C. D. and Longoni, D. and Fioredda, F. and Locatelli, F. and Yaniv, I. and Wachowiak, J. and Donadieu, J. and Lawitschka, A. and Bierings, M. and Wlodarski, M. and Corbacioglu, S. and Bonanomi, S. and Samarasinghe, S. and Leblanc, T. and Dufour, C. and Dalle, J-H (2015) Recommendations on hematopoietic stem cell transplantation for inherited bone marrow failure syndromes. BONE MARROW TRANSPLANTATION, 50 (9). pp. 1168-1172. ISSN 0268-3369, 1476-5365

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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.

Item Type: Article
Uncontrolled 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;
Subjects: 600 Technology > 610 Medical sciences Medicine
Divisions: Medicine > Lehrstuhl für Kinder- und Jugendmedizin
Depositing User: Dr. Gernot Deinzer
Date Deposited: 11 Jun 2019 12:58
Last Modified: 11 Jun 2019 12:58
URI: https://pred.uni-regensburg.de/id/eprint/4883

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