Validity and responsiveness of Patient-reported outcomes Common Terminology Criteria for Adverse Events in the ovarian cancer cohort of the FICOG VIP study

Abstract Background Use of patient-reported outcomes (PROs) is recommended by regulatory authorities for improving patient care. A multicenter Italian study tests the psychometric properties of the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) across cancer types in Italy. Here the analysis of the ovarian cancer (OC) cohort. Methodology A PRO-CTCAE list including 122 items (78 symptomatic adverse events [AEs]), reduced to 78 items (43 symptomatic AEs), was administered to patients with OC on anticancer treatment, at baseline (visit 1) and after 2–6 weeks (visit 2). Convergent validity with the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire - ovarian cancer module (EORTC QLQ-OV28) and Hospital Anxiety and Depression Scale (HADS) was assessed by Pearson correlation. Known-groups validity and responsiveness were tested with Eastern Cooperative Oncology Group performance status (ECOG PS 0 vs. > 0) and Patient Global Impression of Change (PGIC) scale as anchors. Results From July 2019 to March 2024, 214 patients filled PRO-CTCAE, 210 OV28 and 209 HADS at visit 1; 192 and 182 filled PRO-CTCAE and PGIC at visit 2, respectively. Median age (IQR) was 60.8 (53.0–68.8) yrs and 88.3% had PS 0. Convergent validity analysis showed high correlation ( r ≥ 0.50) of PRO-CTCAE hot flushes, hair loss, hand/foot neuropathy, dysgeusia, bloating, abdominal pain, muscle pain, vaginal dryness, heartburn, joint pain, sadness, depression and fatigue with selected OV28 anchors. Anxiety, depression and sadness had medium to large correlation with HADS (r ranging from 0.48 to 0.66). Known-group validity showed small to large correlations (Cohen coefficients ≥ 0.20) with PS for several items. Patients classified as worsened according to the PGIC showed greater deterioration in multiple PRO-CTCAE symptom domains. Conclusions Psychometric properties of PRO-CTCAEs indicate good construct validity and responsiveness, supporting their role in cancer care.