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

R Peffault de Latour (2015) Bone Marrow Transplantation                                          

Link to abstract:

This report summarizes the recommendations for transplanting children with IBMF including indications for HSCT, timing, stem cell source and conditioning regimen for inherited bone marrow failure syndromes. HSCT remains the only curative treatment option for disturbances of hematopoiesis in IBMFS. Prospective international clinical trials are urgently required in order to enhance the management of these rare disorders, and in time, lead to improved outcomes.