Improving quality of health-related quality of life (HRQOL) reporting in phase III randomized controlled trials (RCTs) of metastatic urothelial carcinoma (mUC).

Abstract

467 Background: HRQOL data are increasingly used to guide patient care and health policy. However, their utility can be compromised by inadequate quality of HRQOL reporting (QHR). We aimed to systematically evaluate QHR in phase III RCTs of mUC. Methods: A comprehensive systematic review following PRISMA guidelines identified published manuscripts of phase III RCTs of mUC in English between 1985 - 2019. Supplementary material, references and companion publications were reviewed. QHR was quantified using: 1) 2013 CONSORT-PRO extension (CPE), and 2) 2003 Minimum Standard Checklist for Evaluating HRQOL Outcomes in Cancer Clinical Trials (MSC). Both scores range from 0-11, with higher scores indicating higher QHR. QHR is “probably robust” if MSC score is ≥8 and all 3 mandatory items (baseline compliance, missing data, and psychometric properties) are reported; “limited” if MSC score is 5-7; and “very limited” if MSC score is ≤4. Results: HRQOL data was collected in 7/21 (33%) mUC phase III RCTs. All reported HRQOL data as secondary/exploratory endpoints. Generic instruments included EORTC QLQ-C30 alone (n=3) or combined with EQ-5D (n=2)/McGill pain questionnaire (n=1). Disease-specific FACT-Bl was used in 1 RCT. Both checklists showed strong correlation (Spearman coefficient 0.94, p=0.001). QHR was often limited, however has improved in recent years (Table). No RCT stated HRQOL-specific hypotheses. Only 1 reported instrument validity and mode of administration. Few provided domain specific results (n=2) and statistical methods for handling missing data (n=3). Implications for clinical practice were only discussed in 3 RCTs. Conclusions: QHR has improved over time however many critical methodological deficiencies remain in mUC phase III RCTs. The use of standard checklists are encouraged to further enhance QHR and promote high quality HRQOL research. [Table: see text]