In this installment of the “How I treat …” series in Blood, the author outlines his treatment algorithm for patients with severe aplastic anemia (SAA), using 2 case studies to illustrate the benefits and limitations of immunosuppressive therapy (IST) and hematopoietic cell transplantation (HCT).
Dr. Andrea Bacigalupo reviews research showing how anabolic steroids and growth factors such as thrombopoietin analogs can have a role in supplementing IST and may improve response rates. Two major drawbacks of IST are discussed: a failure of patients to respond to IST and the development of late clonal disorders.
The time between diagnosis and treatment, as well as patient age are strong predictors of survival. After diagnosis, the author suggests that HLA typing of the patient and his/her family should be one of the first interventions for patients with newly diagnosed SAA, because only a portion of patients receiving IST will achieve transfusion independence, or complete recovery of peripheral blood counts.
In the author’s treatment algorithm, a matched sibling donor remains the best choice for patients undergoing HCT, although he notes that unrelated donor transplants “have produced survival comparable to matched sibling donor grafts, though with increased risk of GVHD.”
Dr. Bacigalupo advises that although early results seem encouraging, haploidentical transplants should be considered experimental and are indicated in patients with SAA lacking a suitably matched donor or cord blood unit.