Table_2_Refining patient selection for next-generation immunotherapeutic early-phase clinical trials with a novel and externally validated prognostic nomogram.docx

IntroductionIdentifying which patient may benefit from immunotherapeutic early-phase clinical trials is an unmet need in drug development. Among several proposed prognostic scores, none has been validated in patients receiving immunomodulating agents (IMAs)-based combinations.Patients and methodsWe retrospectively collected data of 208 patients enrolled in early-phase clinical trials investigating IMAs at our Institution, correlating clinical and blood-based variables with overall survival (OS). A retrospective cohort of 50 patients treated with IMAs at Imperial College (Hammersmith Hospital, London, UK) was used for validation.ResultsA total of 173 subjects were selected for analyses. Most frequent cancers included non-small cell lung cancer (26%), hepatocellular carcinoma (21.5%) and glioblastoma (13%). Multivariate analysis (MVA) revealed 3 factors to be independently associated with OS: line of treatment (second and third vs subsequent, HR 0.61, 95% CI 0.40-0.93, p 0.02), serum albumin as continuous variable (HR 0.57, 95% CI 0.36–0.91, p 0.02) and number of metastatic sites (

引言：在药物研发领域，识别哪些患者可能从免疫治疗早期临床试验中获益，是一项尚未满足的需求。在众多提出的预后评分方法中，尚无一种在接受免疫调节剂（IMAs）联合治疗的病人中得到验证。研究方法：本研究回顾性收集了我院208名接受免疫调节剂早期临床试验患者的数据，将临床和血液指标与总生存期（OS）相关联。以伦敦英国帝国理工学院（汉默史密斯医院）治疗的50名接受IMAs治疗的病人组成的回顾性队列用于验证。研究结果：共选择173名受试者进行分析。最常见的癌症包括非小细胞肺癌（26%）、肝细胞癌（21.5%）和胶质母细胞瘤（13%）。多因素分析（MVA）揭示了3个与OS独立相关的因素：治疗线（第二和第三线与后续线相比，HR 0.61，95% CI 0.40-0.93，p 0.02）、血清白蛋白作为连续变量（HR 0.57，95% CI 0.36–0.91，p 0.02）以及转移灶数量（HR 0.57，95% CI 0.36–0.91，p 0.02）。