(1) Background: Quality of life (QoL) assessment is a crucial aspect for patients diagnosed with cancer. Over the years, different tools have been developed to measure QoL, both generic and pathology specific, but the inclusion of quality of life among other indicators of efficacy in randomized controlled trials (RCTs) remains a controversial issue. In this review, we aim to review the frequency and modality of QoL assessment in RCTs, enrolling patients diagnosed with mesenchymal tumors. (2) Methods: An electronic literature search of bone and soft tissue sarcoma and GIST-related RCTs published between January 2000 and December 2023 was performed by two independent reviewers using PubMed. English-language phase II and III clinical trials enrolling at least more than 15 patients were included, regardless of the disease stage. Studies involving patients under the age of 18 years or for which the full text was not available were excluded. For each study, data regarding the journal and year of publication, the study design, the primary objective, and the evaluation of quality of life as an endpoint with any type of patient-reported outcomes used were extracted. (3) Results: Among the 742 publications screened, 171 resulted eligible. QoL assessment was listed among the endpoints in 35 trials and QoL results were reported in 29 primary publications. In these trials, 16 included patients with soft tissue sarcomas, 8 Kaposi sarcomas, 6 GIST, and 3 desmoid tumors. Among all the trials included, 10.4% on an adjuvant/neoadjuvant setting and 24.4% on a metastatic setting included QoL as an endpoint. The proportion of trials, including QoL, was variable over time, as follows: 16.9% of trials in 2000–2014 vs. 23.4% in 2015–2023. In 35 trials, including QoL endpoints, 27 had a superiority design and 25 reported a positive result. In the majority of trials (80%), the tools for QoL assessment were generic and those mostly used were the EORTC QLQ-C30, the EQ-5D questionnaire, and the modified Brief Pain Inventory–Short Form. (4) Conclusions: Quality of life has not been assessed or published in many phase II and III trials, despite an improvement over time. QoL evaluation in RCTs should be considered even more carefully in patients with rare tumors, where the low number of patients who can be enrolled makes it difficult to draw statistically significant conclusions on the effectiveness of treatments.
(1) 背景：生活质量评估是癌症患者诊疗过程中的关键环节。多年来，已开发出通用型及疾病特异性的多种生活质量评估工具，但在随机对照试验中将生活质量纳入疗效评价指标仍存在争议。本综述旨在系统分析间叶组织肿瘤相关随机对照试验中生活质量评估的实施频率与模式。(2) 方法：由两位独立评审员通过PubMed数据库检索2000年1月至2023年12月期间发表的骨与软组织肉瘤及胃肠道间质瘤相关随机对照试验。纳入标准为：英文发表的II/III期临床试验、入组患者≥15例（不限疾病分期）。排除标准为：患者年龄<18岁或无法获取全文的研究。从每项研究中提取期刊信息、发表年份、研究设计、主要研究目的、生活质量终点指标评估及使用的患者报告结局指标类型等数据。(3) 结果：在筛选的742篇文献中，171篇符合纳入标准。其中35项试验将生活质量列为终点指标，29篇主要研究论文报告了生活质量结果。这些试验涉及软组织肉瘤（16项）、卡波西肉瘤（8项）、胃肠道间质瘤（6项）和硬纤维瘤（3项）。在所有纳入试验中，辅助/新辅助治疗试验中10.4%包含生活质量终点，转移性治疗试验中该比例为24.4%。生活质量评估的纳入比例随时间呈现动态变化：2000-2014年为16.9%，2015-2023年升至23.4%。在35项包含生活质量终点的试验中，27项采用优效性设计，25项报告了阳性结果。绝大多数试验（80%）使用通用型评估工具，最常用的是EORTC QLQ-C30量表、EQ-5D问卷及改良版简明疼痛评估简表。(4) 结论：尽管随时间推移有所改善，但多数II/III期试验仍未评估或报告生活质量数据。对于罕见肿瘤的随机对照试验，因可入组患者数量有限导致治疗效果的统计学结论难以确立，更应审慎考虑生活质量评估的价值。
肿瘤(癌症)患者之家