Background: Health-related quality of life (HRQoL) is an important patient-reported outcome for all cancer patients, including adult patients with rare solid cancers. However, current knowledge of HRQoL in this population is limited, which hinders the delivery of personalized care. This review aimed to explore the heterogeneity of HRQoL among adult patients with a solid rare cancer across the ten European Reference Network for Rare Adult Solid Cancers (EURACAN) domains and to summarize the HRQoL measures used in clinical research. Methods: A systematic literature search was conducted to identify all clinical studies assessing HRQoL in adult patients with a solid rare cancer. Four databases (MEDLINE, PubMed, PsycINFO, and Web of Science/Scopus) were searched (February 2023). Results: The search yielded 18,704 articles, of which 1416 articles were fully screened and 463 were eligible for analysis. Of these, 397 studies used generic tools to assess HRQoL, while 270 used tumor-specific instruments. Three EURACAN domains (sarcoma, endocrine tumors, and thoracic tumors) primarily assessed HRQoL using generic questionnaires. Additionally, the rare gynecological tumor, rare male genitourinary, and sarcoma EURACAN domains lacked specific HRQoL measures. Brain, head and neck, and uveal melanoma EURACAN domains used tumor- or domain-specific questionnaires in more than half of the studies. Conclusions: This state-of-the-art literature review shows that HRQoL assessment is gradually becoming more prevalent in adult solid rare cancer research. A combination of generic, tumor-specific, and domain-specific questionnaires across various rare cancer domains has proven effective in capturing a broad range of HRQoL issues. However, many EURACAN domains still lack specific strategies for assessing HRQoL, which limits the ability to fully understand and address patients’ experiences. Future research should prioritize developing comprehensive and robust HRQoL measurement strategies and tools to enable meaningful clinical research and to ensure that the patient voice is incorporated in their clinical care.
背景：健康相关生活质量（HRQoL）是所有癌症患者（包括成人罕见实体瘤患者）报告的重要结局指标。然而，目前对该人群HRQoL的认知有限，这阻碍了个体化医疗的实施。本综述旨在探讨欧洲罕见成人实体瘤参考网络（EURACAN）十大领域中成人罕见实体瘤患者HRQoL的异质性，并总结临床研究中使用的HRQoL测量工具。方法：通过系统性文献检索，筛选所有评估成人罕见实体瘤患者HRQoL的临床研究。检索覆盖四大数据库（MEDLINE、PubMed、PsycINFO及Web of Science/Scopus），检索时间截至2023年2月。结果：初筛获得18,704篇文献，经全文筛查后纳入1416篇，其中463篇符合分析标准。在这些研究中，397项使用通用工具评估HRQoL，270项使用肿瘤特异性工具。EURACAN网络中的三个领域（肉瘤、内分泌肿瘤和胸部肿瘤）主要采用通用问卷评估HRQoL。此外，罕见妇科肿瘤、罕见男性泌尿生殖系统肿瘤及肉瘤领域缺乏特异性HRQoL测量工具。而脑肿瘤、头颈部肿瘤及葡萄膜黑色素瘤领域在超过半数研究中使用了肿瘤或领域特异性问卷。结论：本前沿文献综述表明，HRQoL评估在成人罕见实体瘤研究中正逐渐普及。综合使用通用性、肿瘤特异性及领域特异性问卷已被证明能有效捕捉广泛的HRQoL问题。然而，许多EURACAN领域仍缺乏评估HRQoL的特异性策略，限制了对患者体验的全面理解与应对。未来研究应优先开发全面、稳健的HRQoL测量策略与工具，以推动具有临床意义的研究开展，并确保将患者声音纳入临床诊疗体系。
肿瘤(癌症)患者之家