Journal: British journal of haematology
This study examined how outcome priorities differ among patients with multiple myeloma, clinicians, and randomized controlled trials (RCTs).
Design:
- Mixed-methods:
- Qualitative interviews with 10 patients and 6 clinicians to elicit treatment priorities.
- Systematic review to catalogue endpoints used in myeloma RCTs.
- Quantitative surveys based on these findings, completed by 117 patients and 105 clinicians.
- Statistical comparisons used odds ratios (ORs) with 95% confidence intervals (CIs) to contrast priorities across groups.
Key findings:
- Shared priorities: Both patients and clinicians ranked quality of life (QoL) as the single most important outcome (OR 1.01; 95% CI 0.55–1.87 for patients vs clinicians).
- Clinician priorities: Clinicians were more likely than patients to prioritize:
- Overall survival (OS) (OR 1.92; 95% CI 1.05–3.50).
- Progression-free survival (PFS) (OR 5.37; 95% CI 1.95–14.79).
- Patient priorities: Patients were far more likely to prioritize pain reduction (OR 0.03; 95% CI 0.00–0.23), indicating strong emphasis on symptom control over classical efficacy metrics.
- Comparison with RCT endpoints:
- Patients placed much greater importance on QoL (OR 0.03; 95% CI 0.01–0.07) and pain elimination (OR 0.05; 95% CI 0.02–0.11).
- Trials heavily emphasized PFS (OR 6.33; 95% CI 2.53–15.83) and response rates (OR 17.75; 95% CI 5.56–56.61).
- Clinician–trial alignment: Clinicians’ views were closer to trial designs for PFS, but they still valued QoL and OS more than typical trial endpoints reflect.
Implications for practice and research:
- QoL is a central, shared priority among patients and clinicians but is under-represented as a primary endpoint in myeloma RCTs.
- Clear misalignment: There is a clear misalignment between what patients value (QoL, pain control) and what trials most commonly measure (PFS, response).
- Future trial design: Future myeloma trials should incorporate patient-centred endpoints—especially validated QoL measures and pain outcomes—to better reflect what matters to people living with the disease and to support more meaningful clinical decision-making.