Olverembatinib (HQP1351)-based therapy in adults with relapsed or refractory Philadelphia chromosome-positive acute lymphoblastic leukemia or chronic myeloid leukemia in blast phase: results from a real-world study

作者：Ziyu Wen, Zhi Liu, Xu Ye, Zhiping Fan, Ren Lin, Fen Huang, Li Xuan, Xiaofang Li, Hua Jin, Min Dai, Jing Sun, Xuan Zhou, Qiang Wang, Xiaoli Liu, Qifa Liu, Hongsheng Zhou, Na Xu · 发表于：Frontiers in Immunology · 年份：2025 · DOI：10.3389/fimmu.2025.1546371 · 被引用次数：5 · 研究领域：Chronic Myeloid Leukemia Treatments、Acute Lymphoblastic Leukemia research、Acute Myeloid Leukemia Research

Background Relapsed or refractory Philadelphia chromosome-positive acute lymphoblastic leukemia (R/R Ph + ALL) and chronic myeloid leukemia in the blast phase (CML-BP) are associated with poor prognoses. Olverembatinib (HQP1351), a novel third-generation tyrosine kinase inhibitor (TKI), has shown promising efficacy and safety in clinical trials against nearly all BCR-ABL1 kinase mutations, including T315I . Methods Data were collected and analyzed to evaluate the efficacy and safety of olverembatinib-based therapy for advanced Ph + leukemia. The primary outcome was the overall response rate at 28 days. Secondary outcomes included overall survival (OS), event-free survival (EFS), disease-free survival (DFS), the proportion of patients undergoing allo-HSCT, and adverse events. Results A total of 59 patients participated in the study, including 40 patients with Ph + ALL and 19 with CML-BP. Among them, 36 (61.0%) were men, and 23 (39.0%) were women. The median age was 39 years (interquartile range [IQR], 30–48), and the median follow-up duration was 7.8 months (IQR, 4.1–11.3). A total of 16 (27.1%) and 11 patients (18.6%) had received treatment with two and ≥ 3 prior TKIs, respectively. Additionally, 19 patients (33.9%) had been treated with ponatinib. In a cohort of 19 CML patients, 12 (63.2%) achieved CR/CRi by day 28. Five (26.3%) achieved a complete cytogenetic response with a median duration of 2.9 months, and two (10.5%) achieved a major molecular response with a median dur...