OBJECTIVES: Multiple myeloma (MM) is associated with substantial morbidity and mortality. Treatment selection for patients with MM should consider health-related quality of life (HRQoL), both from disease burden and treatment-related adverse events. This review aimed to explore how patient-reported outcomes (PROs), symptoms, and HRQoL have been evaluated in MM patients, and identify PRO methodology patterns in interventional studies.

METHODS: A targeted literature review (TLR) was conducted using Embase, MEDLINE, and Cochrane to identify relevant publications from 2011–2021 reporting PROs and HRQoL measures in patients with MM at any line of therapy. Interventional and prospective observational trials, qualitative studies, and systematic literature reviews were eligible. The relationship between clinical and PRO endpoints was subjectively explored, particularly regarding pain, fatigue, disease symptoms, functioning, and QoL.

RESULTS: Of 6,525 records identified, 61 interventional studies (115 publications) reporting PROs were included. The most frequently used PRO instruments were: EORTC-QLQ-C30 (n=47), EORTC-QLQ-MY20 (n=27), EQ-5D (n=22), FACT-GOG-NTX (n=7). Emerging measures included: PRO-CTCAE (n=2), FACT-MM (n=1), MySimQ (n=1). Analysis methods varied, with both descriptive (eg, change from baseline, within-patient change) and formal group comparisons (eg, mixed model repeated measures, time-to-event analyses) reported. Time-to-event analyses focused on time to improvement/deterioration, with differing event definitions (eg, distribution-based definitions, first meaningful score change, confirmed or definitive change). Of the 61 studies, 37 exhibited a positively trending clinical primary endpoint (excluding single arm or inferiority trials), 11 of which saw significant improvement from baseline in both the primary and ≥1 PRO endpoint.

CONCLUSIONS: This TLR identified significant heterogeneity in both the PRO instruments and endpoints used. In oncology, and MM specifically, the relationship between PROs and clinical endpoints may vary depending on PRO endpoint definition and analytical method. Measurement strategy should be carefully considered based on key clinical endpoints in interventional studies to accurately capture the patient experience.