Spałek Adrianna, Wieczorkiewicz-Kabut Agata, Zielińska Patrycja, Kopińska Anna, Woźniczka Krzysztof, Koclęga Anna, Butrym Aleksandra, Czyż Jarosław, Helbig Grzegorz
Department of Hematology and Bone Marrow Transplantation, Faculty of Medicine in Katowice, Medical University of Silesia, Katowice, Poland.
Alfred Sokolowski Specialist Hospital in Walbrzych Oncology Support Centre for Clinical Trials, Wałbrzych, Poland.
Expert Rev Hematol. 2025 Sep;18(9):753-760. doi: 10.1080/17474086.2025.2525453. Epub 2025 Jun 27.
Almost 50% of patients with FLT3-ITD AML relapse despite undergoing allogeneic hematopoietic stem cell transplantation (HSCT). FLT-3 inhibitors (FLT3i) can be used in a post-HSCT setting as a relapse prevention.
We retrospectively compared 24 FLT3-mutated AML patients receiving post-HSCT SORA with a control group of 24 FLT3-mutated AML SORA-free individuals. SORA was initiated at a median of 2.9 months after transplantation. Median dosage was 600 mg daily with median treatment duration of 8.8 months. Due to toxicities, 16/24 patients required dose modifications with 6 early SORA withdrawals. After median follow-up of 20.7 months, 1 patient relapsed in SORA-group whereas 9 relapses were observed in the control group (0.004).
SORA maintenance significantly improved OS and RFS probability when compared to the control group (36.3 11.6 months; = 0.01 and 95.5% and 66.7%; = 0.004; respectively). SORA maintenance effectively reduced the risk of death by 93.6% for patients in complete remission with detectable measurable residual disease ( = 0.001). At the last follow-up, 4.2% of patients died in SORA-group when compared with 50% in the control group ( < 0.001).
SORA maintenance after HSCT diminishes the cumulative incidence of relapse as well as prolongs OS and RFS in FLT3-mutated AML.
尽管接受了异基因造血干细胞移植（HSCT），近50%的FLT3-ITD急性髓系白血病（AML）患者仍会复发。FLT-3抑制剂（FLT3i）可在HSCT后用于预防复发。
我们回顾性比较了24例接受HSCT后索拉非尼（SORA）治疗的FLT3突变AML患者与24例未接受SORA治疗的FLT3突变AML患者组成的对照组。SORA在移植后中位2.9个月开始使用。中位剂量为每日600毫克，中位治疗持续时间为8.8个月。由于毒性反应，16/24例患者需要调整剂量，6例患者提前停用SORA。中位随访20.7个月后，SORA组有1例患者复发，而对照组有9例患者复发（P = 0.004）。
与对照组相比，SORA维持治疗显著提高了总生存期（OS）和无复发生存期（RFS）概率（分别为36.3对11.6个月；P = 0.01和95.5%对66.7%；P = 0.004）。对于可检测到微小残留病的完全缓解患者，SORA维持治疗有效降低了93.6%的死亡风险（P = 0.001）。在最后一次随访时，SORA组4.2%的患者死亡，而对照组为50%（P < 0.001）。
HSCT后SORA维持治疗可降低FLT3突变AML的累积复发率，并延长OS和RFS。
