ArticleBritish journal of haematology2026
Patients', clinicians' and research's priorities on important outcomes in multiple myeloma: A mixed-methods study.
Article in British journal of haematology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Authors and funding
14 authors.
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Abstract
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.
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