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

R. Peffault De Latour, C. Peters, B. Gibson, B. Strahm, A. Lankester, C. D. De Heredia, D. Longoni, F. Fioredda, F. Locatelli, I. Yaniv, J. Wachowiak, J. Donadieu, A. Lawitschka, M. Bierings, M. Wlodarski, S. Corbacioglu, S. Bonanomi, S. Samarasinghe, T. Leblanc, C. DufourJ. H. Dalle

Research output: Contribution to journalArticle

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.

Original languageEnglish
Pages (from-to)1168-1172
Number of pages5
JournalBone Marrow Transplantation
Volume50
Issue number9
DOIs
Publication statusPublished - Sep 4 2015

ASJC Scopus subject areas

  • Hematology
  • Transplantation

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    Peffault De Latour, R., Peters, C., Gibson, 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., ... Dalle, J. H. (2015). Recommendations on hematopoietic stem cell transplantation for inherited bone marrow failure syndromes. Bone Marrow Transplantation, 50(9), 1168-1172. https://doi.org/10.1038/bmt.2015.117