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."