Al-Naesan Imad, Krepper Daniela, Sparano Francesco, Sztankay Monika, Efficace Fabio, Giesinger Johannes M
Department of Psychiatry, Psychotherapy, Psychosomatics, and Medical Psychology, Innsbruck Medical University, University Hospital of Psychiatry II, Innsbruck, Austria.
Data Center and Health Outcomes Research Unit, Italian Group for Adult Haematologic Diseases (GIMEMA), Rome, Italy.
Melanoma Res. 2025 Apr 1;35(2):77-86. doi: 10.1097/CMR.0000000000001014. Epub 2024 Dec 11.
The objective of this study was to provide an overview of the current practice of patient-reported outcome (PRO) assessments in trials investigating treatment with BRAF inhibitors in patients with advanced melanomas. In addition, we extracted information on symptomatic adverse events (AEs) reported by clinicians to inform future PRO measurement strategies. For our systematic scoping review, we investigated randomized controlled trials (RCTs) evaluating treatment with BRAF inhibitors that had a primary, secondary or exploratory PRO endpoint and were indexed on PubMed. Two independent reviewers extracted information on general RCT characteristics, clinical results (e.g. survival, treatment response and symptomatic AEs) and the PRO measurement and results. Quality of PRO reporting using the CONSORT-PRO checklist was also assessed. We identified nine RCTs meeting the inclusion criteria, in which PROs were secondary or exploratory endpoints. In all trials but one, PROs were measured with the generic EORTC QLQ-C30 questionnaire. The quality of PRO reporting showed substantial variation across the different types of information, with information on handling of missing data and on PRO hypotheses lacking most frequently. Our analysis identified 29 relevant symptomatic AEs that could be reported directly by patients. Our findings may inform the planning of the PRO component of future RCTs, in particular regarding what symptoms and AEs should be covered by PRO measures to provide a comprehensive assessment of treatment tolerability. Our results also indicate a need for improving the quality of PRO reporting, to maximize the impact of PRO findings in real-word practice.
本研究的目的是概述在晚期黑色素瘤患者中调查BRAF抑制剂治疗的试验中患者报告结局（PRO）评估的当前实践情况。此外，我们提取了临床医生报告的症状性不良事件（AE）信息，以为未来的PRO测量策略提供参考。对于我们的系统综述，我们调查了评估BRAF抑制剂治疗的随机对照试验（RCT），这些试验具有主要、次要或探索性PRO终点，并在PubMed上被索引。两名独立评审员提取了关于一般RCT特征、临床结果（如生存、治疗反应和症状性AE）以及PRO测量和结果的信息。还使用CONSORT-PRO清单评估了PRO报告的质量。我们确定了9项符合纳入标准的RCT，其中PRO是次要或探索性终点。除一项试验外，在所有试验中，PRO均使用通用的欧洲癌症研究与治疗组织QLQ-C30问卷进行测量。PRO报告的质量在不同类型的信息中显示出很大差异，关于缺失数据处理和PRO假设的信息最常缺失。我们的分析确定了29种相关的症状性AE，患者可以直接报告这些AE。我们的研究结果可能为未来RCT的PRO部分规划提供参考，特别是关于PRO测量应涵盖哪些症状和AE，以全面评估治疗耐受性。我们的结果还表明需要提高PRO报告的质量，以最大限度地提高PRO研究结果在实际应用中的影响。
