The role of allogeneic hematopoietic stem cell transplantation in the treatment of rare inherited bone marrow failure syndromes with cancer predisposition
Performing allo-HSCT before the development of advanced clonal evolution and malignant transformation is associated with improved long-term outcomes, suggesting a personalized approach based on the underlying genetic defect, clinical disease course, and evidence of clonal evolution allows for timely transplantation and...