A patient survey indicates quality of life and progression-free survival as equally important outcome measures in multiple myeloma clinical trials

Purpose: To investigate the trade-off between survival outcome and quality of life (QoL) in Multiple Myeloma (MM). Methods We developed a questionnaire that asked patients amongst others, whether they would choose a shortened time of progression-free survival (PFS) in favor of an increased QoL Results Of 194 MM patients who answered this question, 81 (42%) subjects were willing to accept a shorter PFS for better QoL. On the other hand, 92 (47%) preferred a longer PFS at the cost of reduced QoL. Twenty-one patients (11%) indicated to be undecided. Specific features were associated with the two main groups (“in favor QoL” vs “in favor PFS”). Patients who belonged to the “in favor QoL”-group tended to be in more advanced treatment lines when compared to the “in favor PFS-group” (P = 0.0001). Patients who preferred PFS were found to generally be more likely to hand over responsibility to their physicians (P = 0.01). No associations were found for other disease specific conditions including pain, gastrointestinal symptoms, fatigue or infection. Conclusion The results of our study highlight significant differences in patients’ preferences, thus underlining the importance of assessing individual patient needs in determining the endpoints of further research.