中文摘要：

目的分析索拉非尼作为难治复发性FLT3突变阳性急性髓系白血病（AML）患者挽救性治疗策略在异基因造血干细胞移植（allo-HSCT）前后应用的效果。方法16例难治复发性FLT3突变阳性AML患者（10例为移植前难治复发，6例为移植后复发）纳入回顾性研究；索拉非尼应用方法包括索拉非尼联合化疗诱导缓解和缓解后索拉非尼单药维持治疗。结果16例患者中13例经1～2个疗程诱导缓解治疗获完全缓解（CR），包括7例移植前难治复发和6例移植后复发患者。移植后中位随访472（59～1 569）d，12例存活，4例死亡，死亡原因包括复发3例、急性移植物抗宿主病1例。16例患者移植后2年累积总生存率和无病生存率分别为（75.0±10.8）%和（50.5±13.7）%。索拉非尼主要不良反应为皮疹，移植前应用者皮疹发生率低于移植后应用者（30.0%对75.0%, P=0.043）。结论索拉非尼作为难治复发性FLT3突变阳性AML挽救性治疗策略，移植前后应用均能降低移植后患者的复发率，提高生存率。

英文摘要：

Objective To analyze the effect of sorafenib as salvage therapy used before and/or after allogeneic hematopoietic stem cell transplantation （allo-HSCT） in refractory relapsed FLT3-positive acute myeloid leukemia （AML）. Methods A total of 16 patients with refractory relapsed FLT3-positive AML, including 10 refractory relapsed pre-transplantation and 6 relapsed after allo-HSCT, were enrolled in this retrospective study. Sorafenib treatment protocols included sorafenib in combination with chemotherapy inducing remission, and sorafenib monotherapy as mauntenance treatment after complete remission （CR）. Results Thirteen of the 16 patients achieved CR after one or two courses of induction therapy, including 7 refractory relapsed pre-transplantation and 6 relapsed after allo-HSCT. With a median follow up of 472 （range, 59-1 569） days post-transplantation, 12 patients survived and 4 died. Causes of death included leukemia relapse （n=3） and acute graft-versus-host disease （n=1）. The 2-year overall and disease-free survival post-transplantation of the 16 patients were （75.0±10.8）% and （50.5±13.7）% respectively. The main side effect of sorafenib was the skin rash. The incidence of rash was lower in the patients used sorafenib pre-transplantation than those post-transplantation （30.0% vs 75.0%,P=0.043）. Conclusion Sorafenib used as salvage therapy befor and/or after transplantation for refractory relapsed FLT3-positive AML could reduce the relapse rate and improve the survival.