A comprehensive systematic literature review presented at the 2024 American Society of Hematology Annual Meeting & Exposition has uncovered significant disparities between clinical trial data and real-world experiences regarding quality of life impacts in relapsed/refractory multiple myeloma patients.
The study (CRD42023467098) analyzed 175 records from multiple databases, incorporating 34 unique studies with QLQ-C30 outcomes and 19 with QLQ-MY20 outcomes. The findings reveal that clinical trials consistently underestimate the burden of disease on patients' health-related quality of life (HRQOL).
Real-World vs. General Population Comparisons
The research highlighted striking differences between relapsed/refractory multiple myeloma patients and the general population. Global health status/QOL scores showed a meaningful gap (60.0 vs 66.4), exceeding the minimally important difference (MID) threshold of 4 points. Significant disparities were also observed in:
- Physical functioning (71.6 vs 82.9, MID = 5)
- Role functioning (67.5 vs 83.6, MID = 6)
- Social functioning (74.3 vs 88.4, MID = 5)
- Cognitive functioning (82.9 vs 86.8, MID = 3)
Symptom Burden and Financial Impact
The study revealed substantial differences in symptom experiences between multiple myeloma patients and the general population:
- Fatigue scores: 37.6 vs 26.0 (MID = 5)
- Pain levels: 34.8 vs 24.0 (MID = 6)
- Appetite loss: 15.7 vs 7.3 (MID = 5)
- Financial difficulties: 17.3 vs 8.4 (MID = 3)
Clinical Trial vs. Real-World Settings
Analysis of 9,042 patients across 32 studies demonstrated significant differences between real-world and clinical trial settings. Notable disparities included:
- Role functioning: -9.2 difference (95% CI, -13.1 to -5.4)
- Social functioning: -5.6 difference (95% CI, -9.8 to -1.3)
- Fatigue: 6.5 difference (95% CI, 4.3-8.8)
- Dyspnea: 5.1 difference (95% CI, 1.6-8.6)
Dr. Rahul Banerjee from the University of Washington and Fred Hutchinson Cancer Center emphasized the significance of these findings: "The novel finding of a difference between the real-world environment and trial settings suggests that trials may underestimate relapsed/refractory multiple myeloma–associated HRQOL burden. As such, HRQOL improvements in clinical trials may be amplified in real-world settings."
Treatment Line Impact
The research also confirmed that HRQOL burden increases with subsequent lines of treatment, though this observation was limited to four studies. This finding underscores the cumulative impact of multiple therapy lines on patient quality of life, particularly in real-world settings.
These findings have important implications for both clinical practice and future trial design, suggesting that quality of life endpoints may be even more crucial in assessing novel therapies' real-world impact than previously recognized.
