Alternative Donor HCT Better than Immunosuppressive Therapy in Pediatric SAA

This retrospective single-center study of children and adolescents with severe aplastic anemia (SAA) found that upfront hematopoietic cell transplantation (HCT) using alternative donors resulted in significantly better outcomes compared to both salvage HCT and upfront immunosuppressive treatment (IST).

Researchers examined the medical records of 42 patients with SAA who received frontline IST (n=19) or frontline HCT with an alternative donor (n=23) between 1998 and 2012. Alternative donor grafts were unrelated bone marrow or peripheral blood stem cells, unrelated cord blood, and haploidentical.

The estimated event-free survival (EFS) of the frontline HCT group was significantly higher than that of the frontline IST group: 91.3% vs. 30.7%, respectively (p<0.001). The estimated EFS of the frontline HCT group was also higher than that of the 11 patients undergoing salvage HCT after failing upfront IST: 91.3% vs. 50.9% respectively (p=0.015).

The researchers concluded that “frontline alternative donor HCT may be a better treatment option than IST for children and adolescents with SAA who lack a human leukocyte Ag-matched familial donor.”

Choi YB, et al. Bone Marrow Transplant

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