(P 319) PATIENT REPORTED OUTCOME ASSESSMENT AND REPORTING IN RANDOMIZED CLINICAL TRIALS OF SYSTEMIC THERAPY FOR ADVANCED SOFT TISSUE SARCOMAS; PROTOCOL FOR A SYSTEMATIC REVIEW

Objective: Soft tissue sarcomas (STS) are rare, heterogeneous tumors of mesenchymal origin which account for approximately 1% of all cancers in adults(1). Around half of patients with initially localized tumors will eventually develop advanced, incurable disease(2). Hence, one of the main goals of treatment in this patient population is improvement of Health-Related Quality of Life (HRQoL). However, clinical trials in this area typically focus on traditional clinical outcomes such as radiological response, progression free survival and overall survival rather than on patient reported outcomes (PROs). In recent years a more patient-centered approach has been adopted, including incorporation of PROs into clinical trial endpoints. PROs, such as HRQoL, can be combined with survival data to determine the net clinical benefit of a treatment and may enhance patient-centered decision-making. Given the potential relevance of PROs for advanced STS patients, the main objective of this systematic review is to assess to which extent PROs are reported as a primary, secondary or exploratory outcome within randomized controlled trials (RCTs) of systemic treatments for STS patients. We also aim to evaluate the type of PROs which are commonly measured (e.g. HRQoL), tools used to collect PRO data, inclusion of PRO data in primary or secondary publications and the quality of reporting of PRO data.

Methods: The protocol has been written according to the guidelines of the Cochrane Handbook for Systematic Reviews of Interventions and has been registered at the International Prospective Register of Systematic Reviews (PROSPERO; registration number: CRD42020173903) (3). The search will be conducted by a professional librarian and primary investigator using Pubmed, Embase and Google scholar. Only RCTs evaluating systemic treatments in adult patients with advanced STS published between January 2000 and May 2022 and with a minimum of 50 patients will be included. Unpublished RCTs and RCTs evaluating radiotherapy, supportive medications, alternative or complementary medications will be excluded. The software program ‘DistillerSR’ will be used to remove duplicates, store references, mark articles which are eligible, those which are excluded and the reason for exclusion. References of included RCTs will be manually screened in order to select other relevant studies. Two independent reviewers will complete an electronic data extraction form (eDEF) for eligible articles. For each RCT including PRO data basic trial demographics, clinical and PRO characteristics are extracted. A third reviewer will be consulted in case of discrepancies in the two evaluations forms. When consensus is reached a final eDEF will be stored and managed via the PROMOTION database (Patient-Reported Outcome Measurements Over Time In Oncology)(4). This is a database that contains details on cancer RCTs in other cancer types that have included PROs. The quality of reporting PRO data will be assessed by using PRO-specific items from the Consolidated Standards Of Reporting Trials (CONSORT) 2010 statement and the CONSORT-PRO extension (5). Finally, descriptive data will be reported using frequencies and percentages, and where appropriate will be summarized using mean and standard deviation or median and interquartile ranges.

Results: The systematic literature review is currently being conducted and the first results of the search strategy will be presented at CTOS 2022.

Conclusion: This review is the first to explore the extent to which PROs are reported in RCTs evaluating systemic treatment in patients with STS and give more insight in the methodology of PRO data collection and the quality of reporting. The findings from this study could provide guidance for future research, for example whether PRO-data can be integrated with clinical findings to enhance personalized decision making and ultimately improve HRQoL. As the aim of this review is more to give an overview about the use and reporting of PROs in RCTs than evaluating treatment effects no risk of bias assessment will be done. We note there is a risk of bias due to challenges of assessing PROs in unpublished studies. No specific funding for this project was received.