State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.
Tianjin Institutes of Health Science, Tianjin, China.
Front Immunol. 2023 Jul 7;14:1184395. doi: 10.3389/fimmu.2023.1184395. eCollection 2023.
Bruton tyrosine kinase inhibitor (BTKi) has demonstrated substantial efficacy in treating B-cell lymphoproliferative diseases (BLPD). Nonetheless, the significant discontinuation rates due to toxicity or financial reasons cannot be overlooked. In China, empirical evidence on the usage of BTKi remains scarce.
To address this, a retrospective cohort study was conducted focused on 673 Chinese patients with BLPD who underwent at least one month of BTKi therapy.
Median age at BTKi initiation was 60 years. The median duration on BTKi treatment of the whole cohort was 36.4 months. The median post-BTK survival was not reach. BTKi-based treatment was permanently discontinued in 288 (43.8%) patients during follow-up, mostly attributed to progressive disease. Within the first 6 months of BTKi treatment, 76 patients (26.3%) had early treatment discontinuation. Patients with early discontinuation had extreme worse outcome with a median post-discontinuation survival of only 6.9 months. On multivariate analysis, withdrawal BTKi by toxicity and withdrawal BTKi within 6 months retained to be independent predictors of post-BTK survival, after taking account of the response depth, lines of therapy and baseline cytogenetics including 17p deletion. The decision between BTKi monotherapy and combination therapy, along with the preference for first or second-generation BTKi, exerted no significant impact on survival.
These observations contribute valuable real-world insights into the utilization of BTKi in China. We concluded that BTKi is an effective and well-tolerated treatment for long-term use in Chinese patient population. However, it is imperative to stress that a proportion of patients discontinue BTKi early, leading to suboptimal outcomes. This study underscores the importance of adherence to BTKi therapy for improved clinical outcomes in real-world patients.
布鲁顿酪氨酸激酶抑制剂（BTKi）在治疗 B 细胞淋巴增殖性疾病（BLPD）方面显示出显著的疗效。然而，由于毒性或经济原因而导致的停药率不容忽视。在中国，关于 BTKi 使用的经验证据仍然很少。
为了解决这个问题，我们进行了一项回顾性队列研究，该研究纳入了 673 名在中国接受至少一个月 BTKi 治疗的 BLPD 患者。
BTKi 起始时的中位年龄为 60 岁。整个队列的 BTKi 治疗中位持续时间为 36.4 个月。中位 post-BTK 生存未达到。在随访期间，288 名（43.8%）患者因疾病进展而永久性停用 BTKi 治疗。在 BTKi 治疗的前 6 个月内，有 76 名（26.3%）患者早期停药。早期停药的患者预后极差，停药后中位生存时间仅为 6.9 个月。多变量分析显示，毒性导致的 BTKi 停药和 6 个月内停药是 post-BTK 生存的独立预测因素，考虑到反应深度、治疗线数和包括 17p 缺失在内的基线细胞遗传学因素。BTKi 单药治疗与联合治疗的选择，以及第一代或第二代 BTKi 的偏好，对生存没有显著影响。
这些观察结果为 BTKi 在我国的应用提供了有价值的真实世界见解。我们得出结论，BTKi 是一种有效且耐受性良好的治疗方法，可长期用于中国患者人群。然而，必须强调的是，有一部分患者会早期停用 BTKi，导致治疗效果不佳。本研究强调了在真实世界患者中坚持 BTKi 治疗以改善临床结局的重要性。
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