Abstract
BackgroundHaploidentical donor (HID) hematopoietic stem cell transplantation (HSCT) is an alternative treatment method for severe aplastic anemia (SAA) patients lacking suitable identical donors and those who are refractory to immunosuppressive therapy (IST). The current study evaluated the feasibility of upfront haploidentical HSCT in SAA patients.MethodsWe conducted a multicenter study based on a registry database. One hundred fifty-eight SAA patients who underwent upfront transplantation between June 2012 and September 2015 were enrolled.ResultsEighty-nine patients had haploidentical donors (HIDs), and 69 had matched related donors (MRDs) for HSCT. The median times for myeloid engraftment in the HID and MRD cohorts were 12 (range, 9–20) and 11 (range, 8–19) days, with a cumulative incidence of 97.8 and 97.1% (P = 0.528), respectively. HID recipients had an increased cumulative incidence of grades II–IV acute graft-versus-host disease (aGVHD) (30.3 vs. 1.5%, P < 0.001), grades III–IV aGVHD (10.1 vs. 1.5%, P = 0.026), and chronic GVHD (cGVHD) (30.6 vs. 4.4%, P < 0.001) at 1 year but similar extensive cGVHD (3.4 vs. 0%, P = 0.426). The three-year estimated overall survival (OS) rates were 86.1 and 91.3% (P = 0.358), while the three-year estimated failure-free survival (FFS) rates were 85.0 and 89.8% (P = 0.413) in the HID and MRD cohorts, respectively. In multivariate analysis, survival outcome for the entire population was significantly adversely associated with increased transfusions and poor performance status pre-SCT. We did not observe differences in primary engraftment and survival outcomes by donor type.ConclusionsHaploidentical SCT as upfront therapy was an effective and safe option for SAA patients, with favorable outcomes in experienced centers.
Highlights
Haploidentical donor (HID) hematopoietic stem cell transplantation (HSCT) is an alternative treatment method for severe aplastic anemia (SAA) patients lacking suitable identical donors and those who are refractory to immunosuppressive therapy (IST)
Patients Based on the Chinese Bone Marrow Transplantation Registry (CBMTR) registry database, 11 transplant centers in China that used the same protocol for HID HSCT in SAA patients were invited to join this study
In summary, our findings show that upfront HID HSCT is a safe and feasible choice for patients without suitably matched donors
Summary
Haploidentical donor (HID) hematopoietic stem cell transplantation (HSCT) is an alternative treatment method for severe aplastic anemia (SAA) patients lacking suitable identical donors and those who are refractory to immunosuppressive therapy (IST). Severe aplastic anemia (SAA) is a lifethreatening type for which allogeneic hematopoietic stem cell transplantation (HSCT) and immunosuppressive therapy (IST) are the principle treatment modalities. Upfront matched related donor (MRD) HSCT is recommended for patients with SAA younger than 35 years of age [1] and, reportedly, results in long-term survival rates over 80% [2,3,4,5]. According to current therapeutic algorithms, IST with a combination of horse antithymocyte globulin (ATG) and cyclosporin A (CsA) is the preferred first-line treatment for patients without a MRD, and HSCT from a haploidentical donor (HID) should be delayed until one or two courses of IST have failed [1]. IST has a high failure rate, which may impact patient quality of life due to the necessity for further treatment
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