Patients', clinicians' and research's priorities on important outcomes in multiple myeloma: A mixed‐methods study

Journal article

Summary: Research in multiple myeloma increasingly relies on surrogate end‐points to expedite approvals, yet these may not reflect patient priorities. We conducted a mixed‐methods study to identify outcomes valued by patients and clinicians and compare them with end‐points used in randomized controlled trials (RCTs). Interviews with 10 patients and 6 clinicians identified treatment priorities, which, together with end‐points from a systematic review of myeloma RCTs, informed tailored surveys, completed by 117 patients and 105 clinicians. Both groups ranked quality of life (QoL) as most important (odds ratio [OR] 1.01; 95% confidence interval [CI] 0.55–1.87). Clinicians more often prioritized overall survival (OS) (OR 1.92; 95% CI 1.05–3.50) and progression‐free survival (PFS) (OR 5.37; 95% CI 1.95–14.79), whereas patients prioritized pain reduction (OR 0.03; 95% CI 0.00–0.23). Compared with RCT end‐points, patients emphasized QoL (OR 0.03; 95% CI 0.01–0.07) and pain elimination (OR 0.05; 95% CI 0.02–0.11), while trials favoured PFS (OR 6.33; 95% CI 2.53–15.83) and response (OR 17.75; 95% CI 5.56–56.61). Clinicians aligned with trials on PFS but valued QoL and OS more highly. QoL emerged as a shared priority, underscoring the need for patient‐centred trial designs that better capture outcomes meaningful to those living with myeloma.

Authors: Mainou, M; Karagiannis, T; Pagkalidou, E; Liakos, A; Malandrakis, P

• Publication status: Published
• Peer review status: Peer reviewed
• Publisher: Wiley
• Journal: British Journal of Haematology
• Publication date: 2026-02-26
• Acceptance date: 2026-01-08
• DOI: 10.1111/bjh.70396
• EISSN: 1365-2141
• ISSN: 0007-1048
• Language: English
• Keywords: mixed‐methods research; multiple myeloma; outcomes