(1) Background: Exploring real-world data (RWD) regarding immune-related adverse events (irAEs) is crucial to better understand the efficacy and safety of immunotherapy in cancer patient populations excluded from clinical trials. An analysis was conducted to evaluate the presumptive predictive causality between endocrine irAEs and the efficacy of immune check-point inhibitors (ICIs) in metastatic non-small-cell lung cancer (mNSCLC) patients treated in daily practice in Romania. (2) Methods: This was a retrospective cohort study of mNSCLC patients treated with ICIs in a tertiary level hospital in Romania for a period of almost seven years, from November 2017 till July 2024. Endocrine irAEs were well defined as any occurring autoimmune endocrinopathy during ICIs and related to immunotherapy. The hospital endocrinologist (M.C.C.O) diagnosed, treated, and followed these endocrine irAEs in a multidisciplinary approach. We investigated multiple medical variables to assess their impact on ICI effectiveness. Descriptive and statistical analyses were performed. (3) Results: Of 487 cancer patients treated with ICIs, we identified 215 mNSCLC patients who were evaluated for endocrine irAEs and co-medications during ICI therapy. Forty-seven (21.8%) patients experienced endocrine irAEs, thyroiditis being the most frequent and prevalent autoimmune endocrinopathy in 60% of cases. Endocrine irAEs were statistically significant, correlated with ICI efficacy (p= 0.002) for survival analysis. Steroids and proton-pump inhibitors used as co-medication during ICIs had a negative impact on response to therapy. (4) Conclusions: Endocrine irAEs might be considered predictive biomarkers for successful immunotherapy in mNSCLC patients. Co-medication during ICIs had a major influence on the effectiveness of these cutting-edge therapies. RWD plays an important role for oncology daily practice whenever clinical trial evidence is not available to guide decision.
（1）背景：探索免疫相关不良事件（irAEs）的真实世界数据（RWD），对于更好地理解免疫疗法在临床试验排除的癌症患者群体中的疗效和安全性至关重要。本研究旨在评估罗马尼亚日常临床实践中，转移性非小细胞肺癌（mNSCLC）患者接受免疫检查点抑制剂（ICIs）治疗时，内分泌irAEs与疗效之间假定的预测性因果关系。（2）方法：这是一项回顾性队列研究，纳入2017年11月至2024年7月近七年期间，在罗马尼亚一家三级医院接受ICIs治疗的mNSCLC患者。内分泌irAEs被明确定义为ICIs治疗期间发生的、与免疫治疗相关的任何自身免疫性内分泌疾病。医院内分泌科医师（M.C.C.O）采用多学科方法对这些内分泌irAEs进行诊断、治疗和随访。我们调查了多个医疗变量以评估其对ICI疗效的影响，并进行了描述性和统计学分析。（3）结果：在487例接受ICIs治疗的癌症患者中，我们确定了215例mNSCLC患者，对其在ICI治疗期间的内分泌irAEs和合并用药情况进行了评估。47例（21.8%）患者发生了内分泌irAEs，其中甲状腺炎是最常见和普遍的自身免疫性内分泌疾病，占病例的60%。生存分析显示，内分泌irAEs与ICI疗效具有统计学显著相关性（p=0.002）。在ICIs治疗期间，使用类固醇和质子泵抑制剂作为合并用药对治疗反应产生了负面影响。（4）结论：内分泌irAEs可能被视为mNSCLC患者免疫治疗成功的预测性生物标志物。ICIs治疗期间的合并用药对这些前沿疗法的有效性有重要影响。在缺乏临床试验证据指导决策时，RWD对肿瘤学的日常临床实践起着重要作用。
肿瘤(癌症)患者之家