Allogeneic HCT should be considered as first-line treatment for SAA

Researchers from the Fred Hutchinson Cancer Research Center recommend revisions to the initial treatment guidelines for patients newly diagnosed with severe aplastic anemia (SAA). Based on significant improvements in long-term survival, researchers suggest allogeneic hematopoietic cell transplantation (HCT) be considered the first-line therapy for SAA.

Because of the long-term risk of relapse and secondary myelodysplastic syndrome or acute myeloid leukemia associated with non-transplant immunosuppressive therapy (IST), HCT with HLA-identical sibling donors, HLA-matched unrelated donors and HLA-haploidentical donors may be preferable treatment options to non-transplant IST. HCT has high cure rates for SAA and a relatively low-risk for graft-versus-host disease. Many patients are living disease-free for decades after HCT.

In preparation for HCT as a first-line therapy, the authors recommend all newly diagnosed patients have HLA typing done before starting treatment, as well as all family members. This allows for a marrow donor to be identified among family members or an unrelated donor and the patient to receive a transplant in a timely manner.

The authors conclude that "implementation of this first-line treatment strategy will provide patients with severe aplastic anemia the best chance of long-term disease-free survival."

Georges GE, et al. Blood Advances