Background: As recommended in the European Society for Medical Oncology (ESMO) guidelines, assessment of health-related quality of life (HRQoL) should be a relevant endpoint in randomized controlled trials (RCTs) testing new anticancer therapies. However, previous publications by our group and others revealed a frequent underestimation and underreporting of HRQoL results in publication of RCTs in oncology. Herein, we systematically reviewed HRQoL reporting in RCTs testing new treatments in advanced prostate, kidney and urothelial cancers and published between 2010 and 2022. Methods: We searched PubMed RCTs testing novel therapies in genitourinary (GU) cancers and published in fifteen selected journals (Annals of Oncology, BMC Cancer, British Journal of Cancer, Cancer Discovery, Clinical Cancer Research, Clinical Genitourinary cancer, European Journal of Cancer, European Urology, European Urology Oncology, JAMA, JAMA Oncology, Journal of clinical Oncology, Lancet, Lancet Oncology and The New England Journal of Medicine). We excluded trials investigating exclusively best supportive care or behavioral intervention, as well as subgroup or post hoc analyses of previously published trials. For each RCT, we investigated whether HRQoL assessment was performed by protocol and if results were reported in the primary manuscript or in a secondary publication. Results: We found 85 eligible trials published between 2010 and 2022. Only 1/85 RCTs (1.2%) included HRQoL among primary endpoints. Of note, 25/85 (29.4%) RCTs did not include HRQoL among study endpoints. HRQoL results were non-disclosed in 56/85 (65.9%) primary publications. Only 18/85 (21.2%) publications fulfilled at least one item of the CONSORT-PRO checklist. Furthermore, 14/46 (30.4%) RCTs in prostate cancer, 12/25 (48%) in kidney cancer and 3/14 (21.4%) in urothelial cancer reported HRQoL data in primary publications. Next, HRQoL data were disclosed in primary manuscripts of 12/32 (37.5%), 5/13 (38.5%), 5/16 (31.3%) and 5/15 (33.3%) trials evaluating target therapies, chemotherapy, immunotherapy and new hormonal agents, respectively. Next, we found that HRQoL data were reported in 16/42 (38%) and in 13/43 (30.2%) positive and negative trials, respectively. Finally, the rate of RCTs reporting HRQoL results in primary or secondary publications was 55.3% (n = 47/85). Conclusions: Our analysis revealed a relevant underreporting of HRQoL in RCTs in advanced GU cancers. These results highlight the need to dedicate more attention to HRQoL in RCTs to fully assess the value of new anticancer treatments.
背景：根据欧洲肿瘤内科学会（ESMO）指南建议，健康相关生活质量（HRQoL）评估应作为测试新型抗癌疗法的随机对照试验（RCT）的关键终点指标。然而，本团队及既往研究表明，肿瘤学领域RCT的发表文献中普遍存在对HRQoL结果的低估与漏报现象。本研究系统回顾了2010年至2022年间发表的针对晚期前列腺癌、肾癌及尿路上皮癌新疗法的RCT中HRQoL的报告情况。 方法：我们检索了PubMed数据库中发表于15种选定期刊（包括《肿瘤学年鉴》《BMC癌症》《英国癌症杂志》《癌症发现》《临床癌症研究》《临床泌尿生殖系统癌症》《欧洲癌症杂志》《欧洲泌尿外科学》《欧洲泌尿肿瘤学》《美国医学会杂志》《美国医学会杂志·肿瘤学》《临床肿瘤学杂志》《柳叶刀》《柳叶刀·肿瘤学》及《新英格兰医学杂志》）的泌尿生殖系统癌症新疗法RCT。排除标准包括：仅研究最佳支持治疗或行为干预的试验，以及对已发表试验的事后分析或亚组分析。针对每项RCT，我们核查了研究方案是否包含HRQoL评估，以及结果是否发表于主论文或二次出版物中。 结果：共纳入2010-2022年间发表的85项符合标准的试验。仅1/85项RCT（1.2%）将HRQoL设为主要终点。值得注意的是，25/85项（29.4%）RCT未将HRQoL纳入研究终点。56/85项（65.9%）主论文未披露HRQoL结果。仅18/85项（21.2%）出版物满足CONSORT-PRO清单至少一项条目。进一步分析显示：前列腺癌试验中14/46项（30.4%）、肾癌试验中12/25项（48%）、尿路上皮癌试验中3/14项（21.4%）在主论文中报告了HRQoL数据。按治疗类型分层：靶向治疗试验中12/32项（37.5%）、化疗试验中5/13项（38.5%）、免疫治疗试验中5/16项（31.3%）、新型激素药物试验中5/15项（33.3%）在主论文中披露了HRQoL数据。阳性结果试验中16/42项（38%）与阴性结果试验中13/43项（30.2%）报告了HRQoL数据。最终，在主论文或二次出版物中报告HRQoL结果的RCT比例为55.3%（47/85项）。 结论：本分析揭示了晚期泌尿生殖系统癌症RCT中普遍存在的HRQoL漏报现象。这些结果强调需要在RCT中加强对HRQoL的关注，以全面评估新型抗癌疗法的临床价值。
肿瘤(癌症)患者之家