目的：本研究旨在评估基于gilteritinib（Gilt）的联合疗法桥接allo-HSCT治疗FLT3-ITD（）R/R AML的安全性和有效性。此外，其目的还在于评估Gilt维持治疗对allo-HSCT后患者预后的影响。方法： 回顾性分析2019年8月至2023年1月苏州大学附属第一医院收治的26例FLT3-ITD（）R/R AML患者的临床资料。该分析包括对所有入组患者的综合完全缓解率（CRc）、总生存（OS）时间、无病生存（DFS）时间和不良事件的评估。结果：共纳入26例FLT3-ITD（）R/R AML患者，其中男性14例，女性12例，中位年龄38（18~65）岁。难治18例，复发8例。 14~21天疗效评价显示完全缓解（CR）率为26.9%（7/26），血液学不完全恢复CR为57.7%（15/26），部分缓解（PR）率为 7.7% (2/26)。 CRc 为 84.6%（22/26），微小残留病（MRD）阴性率为 65.4%。所有患者的12个月累积OS率为79.0%，24个月累积OS率为72.0%。中位 OS 时间尚未确定。中位随访时间为 16.0 个月。对治疗有反应的患者中，12个月累积DFS率为78.0%，24个月累积DFS率为71.0%。中位 DFS 时间尚未确定。接受allo-HSCT的患者的中位OS时间明显长于未接受allo-HSCT的患者（3.3个月，95%CI 2.2-4.3个月，P=0.005）。未确定allo-HSCT后接受或未接受维持治疗的Gilt患者的中位OS时间，但allo-HSCT治疗后接受Gilt维持治疗的患者的OS时间长于allo-HSCT治疗后未接受维持治疗的患者的OS时间（P=0.019）。本研究中FLT3-ITD突变清除率为38.5%，FLT3-ITD突变清除患者的中位OS时间未确定，但显着长于未FLT3-ITD突变清除患者的中位OS时间（15.0个月； P=0.018）。基于Gilt的联合治疗最常见的3级及以上血液学不良事件包括白细胞减少症（76.9%）、中性粒细胞减少症（76.9%）、发热性中性粒细胞减少症（61.5%）、血小板减少症（69.2%）和贫血（57.7%）。 1例患者在allo-HSCT治疗后口服Gilt维持治疗期间出现分化综合征，但治疗后病情好转。结论：以Gilt为基础的联合疗法治疗FLT3-ITD（）R/R AML非常有效。它表现出高 CRc、MRD 阴性率和快速起效，从而显着改善患者的生存率。此外，FLT3-ITD突变的清除率非常高。此外，在allo-HSCT治疗后实施桥接allo-HSCT和后备母猪维持治疗可显着提高患者的生存率。密切监测和管理治疗期间可能发生的任何不良事件至关重要。

Objective: This study aims to evaluate the safety and effectiveness of gilteritinib (Gilt) -based combination therapy bridging allo-HSCT for FLT3-ITD(+) R/R AML. Additionally, it aims to assess the impact of Gilt maintenance therapy on the prognosis of patients after allo-HSCT. Methods: The clinical data of 26 patients with FLT3-ITD(+) R/R AML treated at the First Affiliated Hospital of Soochow University from August 2019 to January 2023 were retrospectively analyzed. The analysis included an assessment of the composite complete remission rate (CRc), overall survival (OS) time, disease-free survival (DFS) time, and adverse events experienced by all enrolled patients. Results: A total of 26 patients with FLT3-ITD(+) R/R AML were enrolled, including 14 men and 12 women with a median age of 38 (18-65) years. A total of 18 cases were refractory, and eight cases were relapsed. The curative effect evaluation conducted between 14 and 21 days showed that the complete remission (CR) rate was 26.9% (7/26), the CR with hematology incomplete recovery was 57.7% (15/26), and the partial response (PR) rate was 7.7% (2/26). The CRc was 84.6% (22/26), and the minimal residual disease (MRD) negativity rate was 65.4%. The 12 month cumulative OS rate for all patients was 79.0%, and the 24 month cumulative OS rate was 72.0%. The median OS time was not determined. The median follow-up time was 16.0 months. Among the patients who responded to treatment, the 12 month cumulative DFS rate was 78.0%, and the 24 month cumulative DFS rate was 71.0%. The median DFS time was not determined. Patients who received allo-HSCT had a median OS time that was significantly longer than those who did not receive allo-HSCT (3.3 months, 95%CI 2.2-4.3 months, P=0.005). The median OS time of patients with or without Gilt maintenance therapy after allo-HSCT was not determined, but the OS time of patients with Gilt maintenance therapy after allo-HSCT treatment was longer than that of patients without Gilt maintenance therapy after allo-HSCT treatment (P=0.019). The FLT3-ITD mutation clearance rate in this study was 38.5%, and the median OS time of patients with FLT3-ITD mutation clearance was not determined but was significantly longer than the median OS of patients without FLT3-ITD mutation clearance (15.0 months; P=0.018). The most common grade 3 and above hematological adverse events of Gilt-based combination therapy included leukopenia (76.9%), neutropenia (76.9%), febrile neutropenia (61.5%), thrombocytopenia (69.2%), and anemia (57.7%). One patient developed differentiation syndrome during oral Gilt maintenance therapy after allo-HSCT treatment, but his condition improved after treatment. Conclusion: The Gilt-based combination therapy is highly effective in treating FLT3-ITD(+) R/R AML. It demonstrates a high CRc, MRD negativity rate, and rapid onset, leading to a significant improvement in patients' survival. Furthermore, the clearance rate of FLT3-ITD mutation is notably high. Additionally, implementing bridging allo-HSCT and Gilt maintenance therapy after allo-HSCT treatment has considerably enhances patients' survival. Closely monitoring and managing any adverse event that may occur during treatment are crucial.