There is substantial progress treating chronic lymphocytic leukemia (CLL) including US Food and Drug Administration (FDA) approvals of several Bruton tyrosine kinase-inhibitors (BTKi) including ibrutinib, acalabrutinib and zanubrutinib [1]. In this Perspective we suggest why analyses of clinical trials of BTKis are challenging, complex and controversial and need to be confronted.

Choosing the appropriate efficacy endpoint for CLL clinical trials is exigent. Some endpoints such as response rates, progression-free survival (PFS), time-to-next therapy (TTNT) and/or undetectable measurable residual disease (uMRD) state are used but are controversial for key reasons we discuss below.

慢性淋巴细胞白血病 (CLL) 的治疗取得了实质性进展，包括美国食品和药物管理局 (FDA) 批准了几种布鲁顿酪氨酸激酶抑制剂 (BTKi)，包括 ibrutinib、acalabrutinib 和 zanubrutinib [1]。在这篇文章中，我们提出了为什么 BTK 临床试验的分析具有挑战性、复杂性和争议性，需要面对。

为 CLL 临床试验选择适当的疗效终点非常迫切。使用了一些终点，例如缓解率、无进展生存期 (PFS)、下次治疗时间 (TTNT) 和/或不可检测的可测量残留病灶 (uMRD) 状态，但由于我们在下面讨论的关键原因而存在争议。