Pharmacy Department, Catalan Institute of Oncology (ICO)-Hospitalet, Institut d'Investigació Biomèdica de Bellvitge (IDIBELL), School of Medicine, Universitat de Barcelona (UB), Barcelona, Spain.
Research Management Unit (Unitat de Gestió de la recerca: UGR), Medical Oncology Department, Catalan Institute of Oncology (ICO), Badalona-Applied Research Group in Oncology (B-ARGO), Germans Trias I Pujol Research Institute (IGTP), Badalona, Spain.
Cancer Med. 2023 Aug;12(16):17112-17125. doi: 10.1002/cam4.6360. Epub 2023 Jul 26.
Medicines in special situations (MSS) refer to off-label or to unlicensed drugs under investigation (compassionate use). Our objectives were to evaluate characteristics and to estimate overall survival (OS), event-free survival (EFS), and the duration of treatment (DT) of MSS used for cancer treatment at a multicentre comprehensive cancer institution.
Retrospective cohort study on adult cancer patients for whom an MSS treatment was requested (January 2011-December 2020). A descriptive analysis was performed and median OS and EFS and 95% confidence intervals (CIs) were estimated. Survival curves were stratified by type of tumor, ECOG (Eastern Cooperative Oncology Group) performance status (PS), age, sex, treatment stage and type of drug (mechanism of action and target).
Treatment was initiated in 2092 episodes (1930 patients) out of 2377 MSS episodes (2189 patients) requested, 33% for hematological treatment and 87% for advanced stage cancer. Median OS (months) was 21.1 (95% CI 19.4-22.7), median EFS was 5.6 (95% CI 5.1-6.0) months, and median DT was 4.5 [0.0; 115.3] months. OS and EFS statistically significantly favored female patients, ECOG PS ≥2 episodes showed worse OS and EFS outcomes (p < 0.0001). Statistically significant differences in survival were found within solid and hematological cancer, disease stage, drug mechanism of action, and type of cancer (p < 0.001) but not for age. Survival outcomes by tumor subtype and drug are presented both globally and separately based on disease stage.
MSS uses are practiced across almost all cancer types, mostly for advanced disease. ECOG PS ≥2, along with advanced disease, was related to worse survival. Information about real-world outcomes is valuable and contributes to better decision-making regarding MSS and our experience in this field could be of interest for other colleagues.
特殊情况下的药物（MSS）是指标签外用药或处于研究阶段的未上市药物（同情用药）。我们的目的是评估多中心综合癌症机构中用于癌症治疗的 MSS 的特征，并估计其总生存期（OS）、无事件生存期（EFS）和治疗持续时间（DT）。
对 2011 年 1 月至 2020 年 12 月期间请求 MSS 治疗的成年癌症患者进行回顾性队列研究。进行描述性分析，并估计中位 OS 和 EFS 及其 95%置信区间（CI）。根据肿瘤类型、ECOG（东部合作肿瘤学组）表现状态（PS）、年龄、性别、治疗阶段和药物类型（作用机制和靶点）对生存曲线进行分层。
在请求的 2377 例 MSS 病例（2189 例患者）中，有 2092 例（1930 例患者）开始治疗，其中 33%用于血液系统治疗，87%用于晚期癌症。中位 OS（月）为 21.1（95%CI 19.4-22.7），中位 EFS 为 5.6（95%CI 5.1-6.0）个月，中位 DT 为 4.5[0.0；115.3]个月。OS 和 EFS 显著有利于女性患者，ECOG PS≥2 患者的 OS 和 EFS 结局更差（p<0.0001）。在实体瘤和血液系统癌症、疾病分期、药物作用机制和癌症类型（p<0.001）内发现了生存方面的统计学显著差异，但与年龄无关。按肿瘤亚型和药物对生存结果进行了全球和基于疾病阶段的单独呈现。
MSS 被广泛应用于几乎所有癌症类型，主要用于晚期疾病。ECOG PS≥2 以及晚期疾病与较差的生存相关。关于真实世界结局的信息是有价值的，并有助于更好地决策 MSS，我们在这一领域的经验可能对其他同事感兴趣。
