代理终结点
慢性淋巴细胞白血病
医学
肿瘤科
临床终点
威尼斯人
生物标志物
微小残留病
内科学
临床试验
疾病
混淆
白血病
酪氨酸激酶抑制剂
免疫学
临床意义
单克隆
终点测定
癌症
总体生存率
生存分析
重症监护医学
摘要
Measurable residual disease (MRD) has established itself as an essential post-treatment biomarker in chronic lymphocytic leukemia (CLL). While achieving undetectable MRD (uMRD) reliably predicts prolonged progression-free survival (PFS) for individual patients across clinical trials, establishing MRD as a universal, trial-level regulatory surrogate endpoint across contemporary targeted therapies remains a formidable biostatistical hurdle. This perspective addresses the "atomistic fallacy" underlying the apparent erosion of trial-level surrogacy observed in aggregate meta-analyses. We demonstrate that the attenuation of trial-level surrogacy stems from fundamental biological effect modification-specifically the distinction between cytostatic therapies (e.g., continuous Bruton tyrosine kinase inhibitors) and cytotoxic, deep-remission regimens (e.g., fixed-duration venetoclax combinations)-as well as operational confounders introduced by modern adaptive trial designs. Reconciling these individual- and trial-level observations requires transitioning from a binary endorsement model to a mechanism-dependent, context-specific framework for surrogate endpoint validation in CLL.
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