Department of Hematology, Kansai Medical University Hospital, 2-3-1 Shinmachi, Hirakata City, Osaka, Japan.
Department of Hematology, Harasanshin Hospital, Fukuoka, Japan.
Int J Hematol. 2024 Nov;120(5):613-620. doi: 10.1007/s12185-024-03832-x. Epub 2024 Aug 21.
Venetoclax was approved for relapsed/refractory chronic lymphocytic leukemia (R/R CLL) and small lymphocytic leukemia (SLL) in Japan in September 2019; however, clinical data in Japanese patients are limited. This all-case post-marketing surveillance assessed efficacy and safety in Japanese patients with R/R CLL/SLL who started venetoclax treatment between November 2019 and August 2020. Overall, the safety and efficacy analysis sets included 129 and 114 patients, respectively. The overall response rate (ORR) was 57.0%; ORRs were higher in patients with versus without concomitant rituximab (65.4% vs. 54.7%), and in patients with 1 versus ≥ 2 prior lines of therapies (72.5% vs. 44.4%). Adverse events (AEs) were reported in 66.7% of patients (86/129); the most common AEs were neutrophil count decreased (22.5%), white blood cell count decreased (7.8%), and tumor lysis syndrome (TLS; 6.2%). AEs of special interest (TLS, myelosuppression, and infection) were manageable in clinical practice in Japan. Venetoclax is efficacious and safe for R/R CLL/SLL patients in the real-world setting in Japan. ClinicalTrials.gov ID: NCT04198415.
维奈托克于 2019 年 9 月在日本获批用于治疗复发/难治性慢性淋巴细胞白血病（R/R CLL）和小淋巴细胞白血病（SLL）；然而，日本患者的临床数据有限。本全病例上市后监测评估了 2019 年 11 月至 2020 年 8 月期间开始接受维奈托克治疗的日本 R/R CLL/SLL 患者的疗效和安全性。总体而言，安全性和疗效分析集分别纳入了 129 例和 114 例患者。总体缓解率（ORR）为 57.0%；与未同时使用利妥昔单抗的患者相比（65.4% vs. 54.7%），以及与仅接受 1 线既往治疗的患者相比（72.5% vs. 44.4%），接受维奈托克治疗的患者 ORR 更高。66.7%的患者（86/129）报告了不良反应（AE）；最常见的 AEs 是中性粒细胞计数减少（22.5%）、白细胞计数减少（7.8%）和肿瘤溶解综合征（TLS；6.2%）。在日本的临床实践中，特别关注的 AEs（TLS、骨髓抑制和感染）是可控的。维奈托克在日本真实世界环境中对 R/R CLL/SLL 患者是有效且安全的。ClinicalTrials.gov ID：NCT04198415。
