Paravathaneni Mahati, Safa Houssein, Joshi Vidhu, Tamil Monica K, Adashek Jacob J, Ionescu Filip, Shah Savan, Chadha Juskaran S, Gilbert Scott, Manley Brandon, Semaan Adele, Jim Heather S L, Kalos Denise, Kim Youngchul, Spiess Philippe E, Chahoud Jad
Department of Hematology and Oncology, H. Lee Moffitt Cancer Center, and Research Institute, Tampa, FL, 33612, USA.
Department of Internal Medicine, Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY, 10467, USA.
EClinicalMedicine. 2024 Jan 5;68:102413. doi: 10.1016/j.eclinm.2023.102413. eCollection 2024 Feb.
Standardized, high-quality PRO data reporting is crucial for patient centered care in the field of oncology, especially in clinical trials that establish standard of care. This study evaluated PRO endpoint design, conduct and reporting methods in FDA approved drugs for GU malignancies.
A systematic review of the FDA archives identified GU cancer drug approvals from Feb 2007 to July 2022. ClinicalTrials.gov and PubMed were used to retrieve relevant data. PRO data was screened, and analytic tools, interpretation methods in the published papers and study protocols were reviewed. Compliance with PRO reporting standards were assessed using PRO Endpoint Analysis Score (PROEAS), a 24-point scoring scale from Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data Consortium (SISAQOL).
We assessed 40 trial protocols with 27,011 participants, resulting in 14 renal cell cancer (RCC), 16 prostate cancer (PC), and 10 urothelial cancer (UC) approvals. PRO data was published for 27 trials, with 23 PRO publications (85%) focusing solely on PRO data, while 4 (15%) included PRO data in the original paper. Median time between primary clinical and secondary paper with PRO data was 10.5 months (range: 9-25 months). PROs were not planned as primary endpoints for any study but 14 (52%) reported them as secondary, 10 (37%) as exploratory outcomes, and 3 (11%) lacked any clarity on PRO data as endpoint. Mean PROEAS score of all GU cancers was 11.10 (range: 6-15), RCC (11.86, range: 6-15), UC (11.50, range: 9-14), and PC (10.56, range: 6-15). None met all the SISAQOL recommendations.
Low overall PROEAS score and delays in PRO data publication in GU cancer drug trials conducted in the past decade emphasize the need for improvement in quality of design and conduct of PRO endpoint in future trials and accelerated publication of PRO endpoints, using standardized analysis, and prespecified hypothesis driven endpoint. These improvements are essential for facilitating interpretation and application of PRO study findings to define patient care.
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标准化、高质量的患者报告结局（PRO）数据报告对于肿瘤学领域以患者为中心的治疗至关重要，尤其是在确立治疗标准的临床试验中。本研究评估了美国食品药品监督管理局（FDA）批准的用于泌尿生殖系统恶性肿瘤的药物中PRO终点的设计、实施和报告方法。
对FDA档案进行系统回顾，确定2007年2月至2022年7月期间批准的泌尿生殖系统癌症药物。使用ClinicalTrials.gov和PubMed检索相关数据。筛选PRO数据，并审查已发表论文和研究方案中的分析工具、解释方法。使用PRO终点分析评分（PROEAS）评估PRO报告标准的依从性，PROEAS是一种24分的评分量表，来自分析患者报告结局和生活质量终点数据联盟（SISAQOL）制定的国际标准。
我们评估了40项试验方案，涉及27011名参与者，批准了14项肾细胞癌（RCC）、16项前列腺癌（PC）和10项尿路上皮癌（UC）。27项试验发表了PRO数据，其中23篇PRO出版物（85%）仅关注PRO数据，而4篇（15%）在原始论文中包含PRO数据。有PRO数据的主要临床论文和次要论文之间的中位时间为10.5个月（范围：9 - 25个月）。没有任何研究将PRO计划作为主要终点，但14项（52%）将其报告为次要终点，10项（37%）作为探索性结局，3项（11%）在PRO数据作为终点方面缺乏任何明确说明。所有泌尿生殖系统癌症的平均PROEAS评分为11.10（范围：6 - 15），RCC为11.86（范围：6 - 15），UC为11.50（范围：9 - 14），PC为10.56（范围：6 - 15）。没有一项符合SISAQOL的所有建议。
在过去十年进行的泌尿生殖系统癌症药物试验中，PROEAS总体评分较低以及PRO数据发表延迟，这强调了未来试验中需要改进PRO终点的设计和实施质量，并加快使用标准化分析和预先设定的假设驱动终点来发表PRO终点。这些改进对于促进PRO研究结果的解读和应用以确定患者治疗至关重要。
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