The role of allogeneic hematopoietic stem cell transplantation in the treatment of rare inherited bone marrow failure syndromes with cancer predisposition
Introduction. Inherited bone marrow failure syndromes (IBMFS) caused by germline pathogenic variants in hematopoietic genes are associated with a high risk of developing myelodysplastic syndrome (MDS) and acute myeloid leukemia. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the only curative tr...