Background: This systematic review and meta-analysis aimed to evaluate the efficacy and safety of Bruton tyrosine kinase inhibitors (BTKis) for central nervous system lymphoma (CNSL). Methods: A systematic review was carried out to identify relevant studies from the PubMed, Embase, Cochrane Library, Web of Science, WanFang, CNKI, and CBM databases. The studies included patients with CNSL who received BTKis and reported the overall response (OR), complete remission (CR), and partial response (PR). An overall effect analysis was performed using STATA 15.0. A random-effects model was utilized to calculate the pooled rates, and 95% confidence intervals (CI) were determined for all outcomes. Results: A total of 21 studies involving 368 patients were included in the meta-analysis. For newly diagnosed CNSL, due to the small simple size, we conducted a quantitative description, and the ORR could reach up to 100%. For relapsed/refractory patients, the pooled ORR was 72% (95% CI: 64–80%, I2= 54.89%,p= 0.00), with a pooled CR and PR of 43% (95% CI: 33–54%, I2= 65.40%,p= 0.00) and 23% (95% CI: 13–35%, I2= 78.05%,p= 0.00), respectively. Most adverse events were hematology-related and generally manageable. Conclusion: BTKis showed acceptable efficacy and safety in treating patients with CNSL. However, large and well-designed trials are still required to confirm BTKis as a treatment for CNSL.
背景：本系统综述与荟萃分析旨在评估布鲁顿酪氨酸激酶抑制剂（BTKis）治疗中枢神经系统淋巴瘤（CNSL）的有效性与安全性。方法：通过系统检索PubMed、Embase、Cochrane Library、Web of Science、万方、中国知网及中国生物医学文献数据库，筛选出纳入BTKis治疗CNSL患者并报告总体缓解率（OR）、完全缓解率（CR）及部分缓解率（PR）的相关研究。使用STATA 15.0软件进行整体效应分析，采用随机效应模型计算合并率，所有结局指标均计算95%置信区间（CI）。结果：共纳入21项研究，涉及368例患者。针对新诊断CNSL患者，因样本量较小进行定量描述，其总体缓解率最高可达100%。对于复发/难治性患者，合并总体缓解率为72%（95% CI: 64–80%, I²= 54.89%, p= 0.00），合并完全缓解率与部分缓解率分别为43%（95% CI: 33–54%, I²= 65.40%, p= 0.00）和23%（95% CI: 13–35%, I²= 78.05%, p= 0.00）。多数不良事件为血液学相关反应，总体可控。结论：BTKis在CNSL治疗中表现出可接受的有效性与安全性，但仍需大规模严谨设计的临床试验以确证其治疗价值。
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