Coniac Simona, Costache-Outas Mariana Cristina, Antone-Iordache Ionuţ-Lucian, Barbu Ana-Maria, Bardan Victor Teodor, Zamfir Andreea, Ionescu Andreea-Iuliana, Badiu Corin
Department of Medical Oncology, Hospice Hope Bucharest, 023642 Bucharest, Romania.
Department of Endocrinology, "Carol Davila" University of Medicine and Pharmacy, 020021 Bucharest, Romania.
Cancers (Basel). 2025 Mar 31;17(7):1198. doi: 10.3390/cancers17071198.
(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 ( = 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：探索关于免疫相关不良事件（irAEs）的真实世界数据（RWD）对于更好地理解免疫疗法在被排除在临床试验之外的癌症患者群体中的疗效和安全性至关重要。进行了一项分析，以评估罗马尼亚日常实践中接受治疗的转移性非小细胞肺癌（mNSCLC）患者内分泌irAEs与免疫检查点抑制剂（ICIs）疗效之间的假定预测因果关系。(2) 方法：这是一项对罗马尼亚一家三级医院近七年来（从2017年11月至2024年7月）接受ICIs治疗的mNSCLC患者的回顾性队列研究。内分泌irAEs被明确界定为ICIs治疗期间发生的任何自身免疫性内分泌病且与免疫疗法相关。医院内分泌科医生（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。我们调查了多个医学变量以评估它们对ICI有效性的影响。进行了描述性和统计分析。(3) 结果：在487例接受ICIs治疗的癌症患者中，我们确定了215例在ICI治疗期间接受内分泌irAEs和联合用药评估的mNSCLC患者。47例（21.8%）患者发生内分泌irAEs，甲状腺炎是最常见的自身免疫性内分泌病，占60%的病例。内分泌irAEs具有统计学意义，在生存分析中与ICI疗效相关（=0.002）。ICI治疗期间用作联合用药的类固醇和质子泵抑制剂对治疗反应有负面影响。(4) 结论：内分泌irAEs可能被视为mNSCLC患者成功免疫治疗的预测生物标志物。ICI治疗期间的联合用药对这些前沿疗法的有效性有重大影响。每当缺乏临床试验证据来指导决策时，真实世界数据在肿瘤学日常实践中发挥着重要作用。
