Wang Zi, Jia Congwei, Wang Xuan, Zhang Yan, Zhou Daobin, Zhang Wei
Department of Hematology, Peking Union Medical College Hospital, Peking Union Medical College, Chinese Academy of Medical Sciences, 1# Shuai-Fu-Yuan, Wang-Fu-Jing, Beijing, 100730, China.
Department of Pathology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
Adv Ther. 2025 Sep 3. doi: 10.1007/s12325-025-03344-y.
Orelabrutinib is a novel Bruton's tyrosine kinase inhibitor with high selectivity and a favorable safety profile. Despite substantial benefits in chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) with orelabrutinib, real-world data remain scarce. This study aimed to evaluate the efficacy and safety of orelabrutinib regimens for CLL/SLL in a real-world setting.
Between June 1, 2018, and September 30, 2024, a total of 63 patients with CLL/SLL who received an orelabrutinib-based regimen were included in this retrospective study. Data on demographics and characteristics, treatment, outcomes, and safety were retrospectively collected and analyzed. Outcomes included overall response rate (ORR), disease control rate (DCR), progression-free survival (PFS), overall survival (OS), and safety.
Of the 63 included patients, 18 (28.6%) patients received orelabrutinib-based regimen as first-line therapy, 33 (52.4%) as second-line therapy, and 12 (19.0%) as third-line therapy. The ORR and DCR were 90.5% (57/63, 95% CI 80.4-96.4) and 96.8% (61/63, 95% CI: 89.0-99.6), respectively. Regarding different treatment lines, ORR was 94.4% (17/18) for first-line therapy, 93.9% (31/33) for second-line therapy, and 75.0% (9/12) for third-line therapy. At a median follow-up of 72.0 months (range: 24.0-216.0), the median PFS and OS were not reached. The 36-month PFS rate was 80.7% (95% CI 61.4-100.0) and the 36-month OS rate was 85.5% (95% CI 67.4-100.0). Thirty-eight (60.3%) patients experienced at least one adverse event (AE) of any grade during orelabrutinib therapy, with no treatment-related deaths observed. Among the 31 patients previously treated with ibrutinib/zanubrutinib, AEs reported during prior therapy mostly resolved or improved in severity after switching to orelabrutinib.
Orelabrutinib was effective and well tolerated for patients with CLL/SLL, providing evidence of clinical benefit in real-world settings.
奥雷巴替尼是一种新型布鲁顿酪氨酸激酶抑制剂，具有高选择性和良好的安全性。尽管奥雷巴替尼在慢性淋巴细胞白血病（CLL）/小淋巴细胞淋巴瘤（SLL）治疗中具有显著益处，但真实世界数据仍然匮乏。本研究旨在评估奥雷巴替尼方案在真实世界中治疗CLL/SLL的疗效和安全性。
在2018年6月1日至2024年9月30日期间，本回顾性研究共纳入63例接受基于奥雷巴替尼方案治疗的CLL/SLL患者。回顾性收集并分析了患者的人口统计学和特征、治疗、结局及安全性数据。结局指标包括总缓解率（ORR）、疾病控制率（DCR）、无进展生存期（PFS）、总生存期（OS）和安全性。
63例纳入患者中，18例（28.6%）接受基于奥雷巴替尼的方案作为一线治疗，33例（52.4%）作为二线治疗，12例（19.0%）作为三线治疗。ORR和DCR分别为90.5%（57/63，95%CI 80.4-96.4）和96.8%（61/63，95%CI：89.0-99.6）。不同治疗线的ORR分别为：一线治疗94.4%（17/18），二线治疗93.9%（31/33），三线治疗75.0%（9/12）。中位随访72.0个月（范围：24.0-216.0），未达到中位PFS和OS。36个月PFS率为80.7%（95%CI 61.4-100.0），36个月OS率为85.5%（95%CI 67.4-100.0）。38例（60.3%）患者在奥雷巴替尼治疗期间发生至少1次任何级别的不良事件（AE），未观察到与治疗相关的死亡。在31例既往接受伊布替尼/泽布替尼治疗的患者中，既往治疗期间报告的AE在换用奥雷巴替尼后大多缓解或严重程度改善。
奥雷巴替尼对CLL/SLL患者有效且耐受性良好，为真实世界中的临床获益提供了证据。
