尽管同种异体造血干细胞移植 (allo-HCT) 以及新型 FLT3 抑制剂在诱导治疗 (midostaurin) 和复发/难治性治疗 (gilteritinib) 中的发展，FLT3-ITD 突变白血病 (FLT3-ITD AML) 仍然是一个挑战对于现代血液学而言。迄今为止，索拉非尼是唯一一种在 HCT 后维持治疗中能够有效改善无进展生存期和总生存期的抑制剂，尽管其在这种情况下的使用迄今为止尚未获得监管机构的批准。我们研究的目的是通过单中心经验评估索拉非尼维持治疗预防 HCT 后 FLT3-ITD AML 早期复发的可行性、安全性和有效性。我们分析了 2017 年至 2023 年间在我们中心接受 HCT 后 2 年索拉非尼维持治疗的 26 名连续患者。从 HCT 到索拉非尼开始的中位时间为 130 天，中位剂量为每天 200 mg。两名 (8%) 和三名 (12%) 患者分别因毒性和疾病复发而停止维持治疗。 8 名（31%）患者终止了 2 年维持治疗并停用索拉非尼，而 13 名患者仍在接受治疗。总体而言，21/26 名患者 (81%) 存活并处于稳定完全缓解状态，两年无病生存率为 83.61%。上次随访时未报告重大长期毒性。我们的现实经验支持使用索拉非尼作为 FLT3-ITD AML 的 HCT 后维持中可行且有效的治疗选择。版权所有 © 2024 Diral, Furnari, Bruno, Greco, Clerici, Marktel, Farina, Mastaglio, Vago, Piemontese 、佩卡托利、科尔蒂、贝尔纳迪、西塞里和卢波-斯坦赫里尼。

Despite allogeneic hematopoietic stem cell transplant (allo-HCT) and the development of novel FLT3 inhibitors in both induction (midostaurin) and in the relapsed/refractory setting (gilteritinib), FLT3-ITD mutated leukemia (FLT3-ITD+ AML) still represents a challenge for modern hematology. Sorafenib is, to this date, the only inhibitor that demonstrated efficacy in improving both progression-free and overall survival as post-HCT maintenance therapy, even if its use in this setting has not been approved so far by regulatory agencies. The aim of our study was to evaluate the feasibility, safety, and efficacy of sorafenib maintenance in preventing early relapse in FLT3-ITD+ AML after HCT in a single-center experience. We analyzed 26 consecutive patients who received post-HCT 2-year maintenance with sorafenib at our center between 2017 and 2023. The median time from HCT to sorafenib start was 130 days, and the median dosage was 200 mg per day. Two (8%) and three (12%) patients discontinued maintenance due to toxicity and disease relapse, respectively. Eight (31%) patients terminated the 2-year maintenance and stopped sorafenib, while 13 patients are still under treatment. Overall, 21/26 patients (81%) are alive and in stable complete remission as outlined by a 2-year disease-free survival of 83.61%. No major long-term toxicity was reported at the last follow-up. Our real-world experience supports the use of sorafenib as a feasible and effective therapeutic option in post-HCT maintenance for FLT3-ITD+ AML.Copyright © 2024 Diral, Furnari, Bruno, Greco, Clerici, Marktel, Farina, Mastaglio, Vago, Piemontese, Peccatori, Corti, Bernardi, Ciceri and Lupo-Stanghellini.