Advances in our understanding of inherited and acquired forms of bone marrow failure present new opportunities and challenges for the evaluation and medical management of these patients. This session will focus on emerging new diagnostic and therapeutic approaches to bone marrow failure.
Dr. Akiko Shimamura will discuss germline and somatic genomic analysis of bone marrow failure. She will review current studies on the use of genomics to inform diagnosis, prognosis, and risk of progression to MDS or AML. The challenges of applying genomics to clinical care will be explored.
Dr. Danielle Townsley will present recent advances in non-transplant medical therapies for bone marrow failure disorders. She will provide an update on clinical trials of eltrombopag to treat aplastic anemia and discuss new data on the use of androgens to treat bone marrow failure disorders.
Dr. Regis Peffault de Latour will review current issues for hematopoietic stem cell transplantation to treat acquired and inherited bone marrow failure disorders. The effect of age on transplantation outcomes, the role of transplantation in PNH, and the prevention of chronic graft versus host disease will be discussed. Emerging strategies such as the role of upfront unrelated donor and alternative donor transplants in acquired aplastic anemia, as well as advances in the treatment of clonal evolution in Fanconi anemia will also be addressed.