(P 186) PATIENT REPORTED OUTCOME ASSESSMENT AND REPORTING IN RANDOMIZED CLINICAL TRIALS OF SYSTEMIC THERAPY FOR ADVANCED SOFT TISSUE SARCOMAS: A SYSTEMATIC REVIEW.

Objective: In advanced soft tissue sarcoma (STS), improvement of health-related quality of life (HRQoL) is a primary goal of treatment. The primary objective of this systematic review is to assess to what extent patient reported outcomes (PROs) are reported as a primary, secondary or exploratory outcome within randomized controlled trials (RCTs) of systemic treatments for STS patients with advanced disease. Secondary objectives are to describe the type of PROs measured (e.g. HRQoL), how often PROs are reported in the primary or secondary publication and, if applicable, the duration between primary and secondary publication, tools used to collect PRO data and the quality of reporting of PRO data.

Methods: A systematic literature search from January 2000 – August 2022 was conducted for phase II/III RCTs evaluating systemic treatments in adult patients with advanced STS. RCTs with less than 50 patients, gastrointestinal stromal tumors or HIV-associated Kaposi sarcoma were excluded. Data was captured through electronic data extraction forms by two independent researchers. A third reviewer was consulted in case of any discrepancies. The quality of PRO reporting was assessed according to the CONSORT (Consolidated Standards of Reporting Trials) PRO criteria extension. The number of RCTs that adhered to each CONSORT PRO item, as well as the total CONSORT PRO adherence score for each RCT were reported.

Results: The literature search resulted in 7294 records of which 59 articles, comprising 43 RCTs, were included (Fig. 1). Of these, only 10 RCTs (23%) also included PROs, as either secondary or exploratory outcomes in 8 and 2 RCTs, respectively. In nearly all RCTs (9/10) PROs were assessed with the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core-30 (EORTC QLQ-C30), in some cases in conjunction with another PRO instrument such as the EQ visual analogue scale (EQ-VAS) (1/10), fatigue-specific FA-13 questionnaires (1/10), the EQ-5D questionnaire (2/10) and the modified Brief Pain inventory-short form (mBPI-sf) (1/10). PROs were reported in the primary (6/10) or secondary publication (1/10) or in both the primary and secondary publication (3/10). The median time between primary and secondary publication was 23 months (range 12-39 months). The median adherence score for the CONSORT-PRO extension was 5.5/14 (range 2/14 – 11/14) with the poorest reported items being: describing the PRO hypothesis and domains (2/10 and 0/10), mode of administration (1/10), strategy for dealing with missing data (4/10), reporting PRO study limitations (4/10), generalizability of results (3/10) and relation with clinical outcomes (4/10) (table 1). The adherence score was higher in publications focusing exclusively on PROs (11/14, 10/14, 11/14 and 11/14).

Conclusion: Although HRQoL is a critical aspect in patients with advanced STS, most RCTs conducted thus far in these patients did not include HRQoL or other types of PROs, and in none of the RCTs identified, PROs were included as a primary endpoint. Despite development of the CONSORT-PRO extension in 2013, quality of PRO reporting in RCTs was extremely poor. Higher quality of PRO reporting is needed to more robustly inform patient care in the setting of advanced STS.