异基因造血干细胞移植治疗41例重型再生障碍性贫血疗效分析

OBJECTIVE: To evaluate the efficacy of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for patients with severe aplastic anemia (SAA). METHODS: A retrospective study was conducted in 41 SAA patients received allo-HSCT from Oct. 2001 to May 2015. There were 27 males and 14 females with median age of 17(2–43) years old. Of them, 24 received matched sibling donor HSCT, 17 received unrelated donor transplantation. RESULTS: Hematopoiesis reconstitution was achieved in 38 patients (92.68%). Median time of neutrophils and platelets implantation was 16(10–57) d and 20(9–83) d in evaluable patients, respectively. Acute GVHD occurred in 13 cases, chronic GVHD in 8 cases, and graft rejection in 5 cases. Median follow-up time was 27(3–154) months. The prospective OS for 3 years was (75.1±8.3)%. Transplantation related mortality was 24.39% (10 cases). Multivariate analysis revealed that grade Ⅱ–Ⅳ aGVHD [P=0.018, OR=27.481 (95% CI 2.377–392.636)] and invasive fungal disease [P=0.021, OR=21.364 (95% CI 1.732–354.185)] were independent risk factors of OS for SAA patients after allo-HSCT. CONCLUSION: Allo-HSCT is an efficient and safe therapy for the patients with SAA, not only for patients with HLA matched sibling donor, but also for those with only HLA matched unrelated donor available. Grade Ⅱ–Ⅳ aGVHD and invasive fungal disease were associated with lower OS rate.