Treatment for patients with severe aplastic anemia by haploidentical allogeneic hematopoietic stem cell transplantation

Abstract

Objective To investigate the efficacy, safety and prevention of complications of haploidentical allogeneic hematopoietic stem cell transplantation (Haplo-HSCT) in patients with severe aplastic anemia (SAA). Methods The clinical data of 8 SAA patients treated with Haplo-HSCT were retrospectively analyzed, with male in 5 cases, female in 3 cases, and an average age of 14 years. The donors of hematopoietic stem cells were patient's parents. The preparative regimen was cyclophosphamide + fludarabine + anti-human lymphocyte immune globulin, and 2 patients combined with total body irradiation. The recipients received cyclosporine, short-term methotrexate and mycophenolate mofetil (MMF) for preventing graft versus host disease (GVHD). The hematopoietic reconstitution, the chimerism rate of donor cells, and the incidence of postoperative complications such as infection, GVHD, liver veno-occlusive disease (VOD), hemorrhagic cystitis (HC), and cytomegalovirus (CMV) were observed. Results Hematopoietic reconstitution was achieved in all the 8 patients, the neutrophil engraftment time was (14.80 ± 3.24) d, and the platelet engraftment time was (15.00 ± 3.42) d. One month after transplantation, all the patients had complete DNA chimerism. Seven patients occurred acute GVHD, including Ⅰ-Ⅱ grade in 5 cases, Ⅲ grade in 1 case, and Ⅳ grade in 1 case. Two patients occurred chronic GVHD, including 1 case with localized GVHD in the oral cavity and 1 case with extensive type chronic GVHD in the whole body skin. No liver VOD or HC occurred. No transplantation-related death occurred at average follow-up time of 8.5 (2 -18) months. Conclusions Haplo-HSCT is safe and effective in patients with SAA. Key words: Anemia, aplastic; Hematopoietic stem cell transplantation; Retrospective studies; Haploidentical