引言：对于复发或难治急性髓系白血病（R/R AML）和髓母细胞肉瘤（MS）的治疗已经存在了几十年的挑战。关于Selinexor与各种标准或强化化疗方案结合治疗R/R AML的研究已经取得了令人鼓舞的结果。本研究旨在评估与Selinexor联合使用的无化疗或低剂量化疗方案对R/R AML和MS患者的疗效和安全性。方法：本研究纳入了2021年10月至2022年8月在同济医院接受无化疗或低剂量化疗方案与Selinexor联合治疗的10例R/R AML或MS患者。主要终点为总体反应率（ORR），次要终点包括完全缓解（CR），具有不完全造血恢复的完全缓解（CRi），部分缓解（PR），移植率和安全性。结果：所有患者均可评估反应，4例患者（40.0％）取得CR，2例患者（20.0％）取得CRi，总的CR/CRi为60.0％。包括PR患者的ORR为80.0％。在Selinexor联合治疗后，5例患者（50.0％）进行了异基因造血干细胞移植（allo-HSCT）。随访结束时，有7例（70.0％）患者仍然存活，3例患者因移植相关并发症或疾病进展而死亡。患者中最常见的非血液学不良事件（AE）为1级或2级无症状低钠血症。结论：无化疗或低剂量化疗方案与Selinexor联合治疗R/R AML是可行和可耐受的，并为患者提供了接受移植的机会。版权所有 © 2023 Tong, Jin, Xu, Su, Li, Li, Peng, Mao, Huang 和 Zhang。

Introduction: Treatment of relapsed or refractory acute myeloid leukemia (R/R AML) and myeloid sarcoma (MS) has presented challenges for decades. Studies on selinexor in combination with various standard or intensive chemotherapy regimens for the treatment of R/R AML have demonstrated promising results. This study aimed to evaluate the efficacy and safety of chemotherapy-free or low-dose chemotherapy regimens with selinexor for R/R AML and MS patients. Methods: Ten patients with R/R AML or MS who received chemotherapy-free or low-dose chemotherapy regimens in combination with selinexor at Tongji Hospital from October 2021 to August 2022 were included in this study. The primary endpoint was overall response rate (ORR) and secondary endpoints included complete remission (CR), CR with incomplete hematological recovery (CRi), partial remission (PR), transplantation rate, and safety. Results: All patients were evaluable for response, achieving CR in four (40.0%) patients and CRi in two (20.0%) patients for a total CR/CRi of 60.0%. The ORR was 80.0% when patients with PR were included. Five (50.0%) patients underwent allogeneic hematopoietic stem cell transplantation (allo-HSCT) after treatment with selinexor-containing regimens. At the end of the follow-up, seven (70.0%) patients were alive, and three patients died of transplant-related complications or disease progression. The most frequently reported nonhematologic adverse events (AEs) in patients were grade 1 or 2 asymptomatic hyponatremia. Conclusion: The chemotherapy-free or low-dose chemotherapy regimens in combination with selinexor for R/R AML are feasible and tolerable and provide an opportunity for patients to receive transplantation.Copyright © 2023 Tong, Jin, Xu, Su, Li, Li, Peng, Mao, Huang and Zhang.