Comparison of Hematopoietic Stem Cell Transplantation Outcomes Using Matched Sibling Donors, Haploidentical Donors, and Immunosuppressive Therapy for Patients With Acquired Aplastic Anemia

Abstract

We retrospectively compared the outcomes of 387 consecutive patients with acquired aplastic anemia (AA) who underwent hematopoietic stem cell transplantation (HSCT) with a fludarabine-based conditioning regimen from matched sibling donors (MSD) (n = 108) or haploidentical donors (HID) (n = 91) and immunosuppressive therapy (IST) (n = 188) from 2014 to 2020 at our hospital. Compared with HID-HSCT, MSD-HSCT had a lower incidence of graft failure (1% vs. 7%, p = 0.062), grade II–IV acute graft versus host disease (aGvHD) (16% vs. 35%, p = 0.001), and mild to severe chronic GvHD (cGvHD) (8% vs. 23%, p = 0.007), but an equivalent incidence of grade III–IV aGvHD (8% vs. 12%, p = 0.237) and moderate to severe cGvHD (3% vs. 9%, p = 0.076). HSCT had superior blood count recovery at 3, 6, and 12 months compared with IST (p < 0.001). The estimated 5-year overall survival (OS) of the MSD, HID, and IST groups were 86%, 72%, and 79% (p = 0.02), respectively; accordingly, the failure-free survival (FFS) rates were 85%, 68%, and 56%, respectively (p < 0.001). For patients aged ≤40 years, the OS rate was still significantly superior for MSD-HSCT receipients compared to HID-HSCT receipients (89% vs. 76%, p = 0.024) while the HID-HSCT recipients showed similar OS (76% vs. 78%, p = 0.166) but superior FFS (p = 0.047) when follow-up was longer than 14.5 months in contrast to IST. In a multivariate analysis, HID-HSCT and a conditioning regimen that included busulfan were adversely related to OS among patients who received allografts. In conclusion, MSD-HSCT was the frontline choice for patients with severe AA aged ≤40 years, while HID-HSCT was as effective as IST for patients without an MSD.

The prognostic impact of previously infectious complications on allogeneic hematopoietic stem cell transplantation for patients with severe aplastic anemia: A single-center, retrospective study

“…Conditioning regimens, GVHD prophylaxis, and infection prophylaxis were consistent with our previous studies ( 4 , 7 ).…”

“…It has the advantages of universal and immediate availability of donors. In our previous study, we showed that HID-HSCT had a comparable OS but superior FFS compared with IST ( 4 ). Xu et al ( 2 ) and Liu et al ( 3 ) also reported similar results, demonstrating that HID-HSCT was superior to IST in terms of FFS.…”

“…This difference may be ascribed to HID-HSCT as salvage therapy and a longer interval from diagnosis to therapy in our patients (5 months vs . 1.5 months) ( 4 ).…”

“…The cornerstone of treatment is considered to reserve normal hematopoiesis by modulating the immune environment through intensive immunosuppressive therapy (IST) or regenerating new blood cells by allogeneic hematopoietic stem cell transplantation (allo-HSCT). Although IST is equal to allo-HSCT in terms of overall survival (OS), its disadvantage is significantly lower failure-free survival (FFS), and treatment failures include no hematological response, relapse, and clonal evolution (1)(2)(3)(4); however, the drawbacks of allo-HSCT are high transplant-related mortality (TRM) and potential long-term complications. Patients with previous infections may also have an influence on their choice of therapy.…”

Long-term survivors demonstrate superior quality of life after haploidentical stem cell transplantation to matched sibling donor transplantation

“…The institutional review board approved all study procedures and forms. The GVHD prophylaxis was cyclosporin (CSA) or tacrolimus (FK506) (from day − 1) based [17][18][19]. Methotrexate was used for 3 doses (days + 1, + 3 and + 6) in MSD-HSCT and 4 doses (days + 1, + 3, + 6 and + 11) for HID HSCT.…”