Treatment of inherited bone marrow failure syndromes beyond transplantation

Abstract excerpt

Despite significant progress in transplantation by the addition of alternative hematopoietic stem cell sources, many patients with inherited bone marrow failure syndromes are still not eligible for a transplant. In addition, the availability of sequencing panels has significantly improved diagnosis by identifying cryptic inherited cases. Androgens are the main nontransplant therapy for bone marrow failure in...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Acute Myeloid Leukemia Research
• Share to Blood disorders and treatments
• Share to Erythrocyte Function and Pathophysiology