Immunosuppressive therapy versus haploidentical transplantation in adults with acquired severe aplastic anemia

Our study aimed to compare treatment outcomes between hematopoietic stem cell transplantation (HSCT) from haploidentical donors (HID) and immunosuppressive therapy (IST) in adults with acquired severe aplastic anemia (SAA). The medical records of 113 SAA adults who received IST, including rabbit ATG and cyclosporin (N = 37), or HID HSCT (N = 76) within 6 months of diagnosis at two institutions were retrospectively reviewed. Estimated 8-year overall survival (OS) was comparable between the IST and HID HSCT groups (75.6 vs. 83.7%, respectively, P = 0.328), but failure free survival (FFS) was significantly lower in IST group than HID HSCT group (38.5 vs. 83.7%, respectively, P = 0.001). Furthermore, a significant improvement in FFS was observed with HSCT over IST in patients under 40 years old. At the last follow-up, patients in HSCT group achieved better Karnofsky Performance Status (KPS) than those in IST group (100 [20-100] vs. 90 [20-100], P = 0.002). In terms of blood count, 83.1% (54/65) of patients in HSCT group showed complete recovery compared to only 38.2% (13/34) in IST group (P < 0.001). These data suggest that HID HSCT could be an effective alternative treatment option for SAA adults, and additional prospective studies are necessary.