米哚妥林联合强化化疗治疗核心结合因子白血病的疗效：一项II期临床试验
Efficacy of Midostaurin Combined With Intensive Chemotherapy in Core Binding Factor Leukemia: A Phase II Clinical Trial.
作者信息
Cairoli Roberto, Gatti Arianna, Grillo Giovanni, Stefanucci Marta Rachele, Di Camillo Barbara, Fumagalli Monica, Krampera Mauro, Nadali Gianpaolo, Zappasodi Patrizia, Borlenghi Erika, Todisco Elisabetta, Ubezio Marta, Bernardi Massimo, Molteni Alfredo, Basilico Claudia, Turrini Mauro, Greco Rosa, Mancini Valentina, Riva Marta, Bernasconi Davide Paolo, Brando Bruno, Veronese Silvio Marco, Beghini Alessandro
机构信息
Department of Hematology, ASST Grande Ospedale Metropolitano Niguarda, Milano, Italy.
Department of Medicine and Surgery, University of Milano Bicocca, Milano, Italy.
出版信息
Am J Hematol. 2025 Feb;100(2):346-349. doi: 10.1002/ajh.27547. Epub 2024 Dec 10.
Samples from 34 adult patients newly diagnosed with core binding factor leukemia (CBFL) were collected both at the time of diagnosis and at relapse and were centrally analyzed. Eligible patients received either standard induction CT known as "3 + 7" or an equivalent regimen, according to the recruiting center's policy. Patients who achieved CR or CRi received 3 courses of high-dose ARA-C (Cytarabine) 3000 mg/m every 12 h on days 1, 3, and 5, along with midostaurin at the dose of 50 mg b.i.d from Day 8 to Day 21 as part of consolidation therapy. Following the completion of the consolidation phase, patients received midostaurin as a monotherapy at the dose of 50 mg b.i.d. for 1 year as continuation therapy. The CR rate was 97%; we recorded an OS rate of 73.52% and a DFS rate of 48.4% for the entire cohort. The RI was 38.8% in the CBFB::MYH11 and 66.6% in the RUNX1::RUNX1T1 group. MRD (Measurable Residual Disease) was assessed by RQ-PCR at 10 time points throughout the study, as indicated by arrows.
收集了34例新诊断为核心结合因子白血病（CBFL）的成年患者在诊断时和复发时的样本，并进行集中分析。根据招募中心的政策，符合条件的患者接受了称为“3+7”的标准诱导化疗或等效方案。达到完全缓解（CR）或血细胞计数部分缓解（CRi）的患者接受3个疗程的大剂量阿糖胞苷（Cytarabine），剂量为3000mg/m²，每12小时一次，在第1、3和5天给药，同时从第8天至第21天给予米哚妥林，剂量为50mg，每日两次，作为巩固治疗的一部分。巩固阶段完成后，患者接受米哚妥林单药治疗，剂量为50mg，每日两次，持续1年作为维持治疗。CR率为97%；我们记录了整个队列的总生存率（OS）为73.52%，无病生存率（DFS）为48.4%。CBFB::MYH11组的复发率（RI）为38.8%，RUNX1::RUNX1T1组为66.6%。如箭头所示，在整个研究过程中的10个时间点通过逆转录定量聚合酶链反应（RQ-PCR）评估微小残留病（MRD）。
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