HRQOL Decreases as Treatment Lines Increase in Relapsed/Refractory Multiple Myeloma

A recent study has highlighted a critical aspect of treatment for patients with relapsed or refractory multiple myeloma: the impact on health-related quality of life (HRQOL). The research, a systematic review of literature, shows that with each additional line of therapy, HRQOL can actually decline, not improve as might be expected.

How Does HRQOL Compare Between Myeloma Patients and the General Population?

When comparing the global health status/QOL score between patients with relapsed/refractory multiple myeloma and the general population, the difference was significant. Patients with myeloma scored a 60.0, while the general population scored a 66.4, representing a minimally important difference (MID) of 4 points.

This pattern holds across various functional scales assessing physical functioning, cognitive functioning, role functioning, and social functioning. For instance, patients with multiple myeloma scored 71.6 in physical functioning versus 82.9 in the general population (MID = 5), and 74.3 versus 88.4 in social functioning (MID = 5).

When it comes to symptom scales, the gap is even more pronounced. Patients with myeloma reported higher symptoms such as appetite loss (15.7 vs. 7.3, MID = 5), fatigue (37.6 vs. 26.0, MID = 5), and pain (34.8 vs. 24.0, MID = 6) compared to those in the general population. Financial difficulties also affected myeloma patients more, with a score of 17.3 compared to 8.4 in the general population (MID = 3).

Methodology and Key Findings

To draw these conclusions, researchers conducted a systematic review of 175 records from MEDLINE, Embase, and the Cochrane Central Register of Controlled Trials (CENTRAL). They included any observational real-world evidence or clinical trials reporting HRQOL outcomes in myeloma patients treated with pharmacological interventions.

The study utilized the European Organization for Research and Treatment of Cancer’s (EORTC) Quality of Life Questionnaires, specifically the 30-item Core Quality of Life Questionnaire (QLQ-C30) and the 20-item EORTC Myeloma Module Questionnaire (QLQ-MY20). By synthesizing data from over 30 unique studies, investigators were able to compare real-world evidence with clinical trial outcomes.

One of the most significant findings was the discrepancy between real-world evidence and clinical trial data. For instance, global health status scores demonstrated a -2.5 difference with real-world evidence compared to clinical trials (95% CI, -4.5 to -0.5). Similar differences were observed in other domains, including role functioning, emotional functioning, cognitive functioning, and social functioning.

Specifically, fatigue scores, from 28 trials with 7831 patients, showed a difference of 6.5 between real-world evidence and clinical trials (95% CI, 4.3-8.8). Symptoms like nausea, vomiting, dyspnea, and constipation also had notable differences between the two settings.

The Implications of These Findings

Rahul Banerjee, MD, assistant professor in the Division of Hematology and Oncology at the University of Washington and assistant professor in the Clinical Research Division at the Fred Hutchinson Cancer Center, provided insight into the significance of these findings. “By quantifying pre-treatment HRQOL burden in both trial and real-world settings, our study offers a benchmark for contextualizing patient burden as novel therapies for relapsed/refractory multiple myeloma are developed,” Banerjee wrote.

“The key discovery that there is a difference between real-world data and clinical trials suggests that trials may underestimate the quality-of-life burden associated with relapsed/refractory multiple myeloma. This implies that improvements in HRQOL seen in clinical trials could be amplified when transitioning to real-world settings, underscoring the importance of HRQOL as a crucial endpoint for evaluating new treatments.”

Increasing Treatment Lines and Rising Burden

Another striking finding from the study was that the HRQOL burden increased with each additional line of treatment. Although this was observed in only four studies, it substantiates the growing burden faced by patients as treatments escalate in intensity.

These findings underscore the critical need for both clinicians and researchers to address quality-of-life considerations alongside traditional measures of efficacy and safety in the treatment of relapsed/refractory multiple myeloma. By doing so, they can better support patients through the complexities of their illness.

As novel therapies continue to evolve and become more accessible, understanding the true impact of these treatments on HRQOL is essential. This study not only provides valuable data for patients and providers but also highlights the importance of real-world evidence in guiding the development and application of future treatments.

Conclusion

In summary, this study reveals significant differences in HRQOL between myeloma patients and the general population, especially as treatment lines increase. Real-world evidence suggests that the burden on HRQOL may be higher than indicated in controlled clinical trials. These insights emphasize the need for a comprehensive approach to patient care in relapsed/refractory multiple myeloma, giving due consideration to both therapeutic efficacy and patient quality of life.

We invite you to share your thoughts and experiences with our community. Whether you are a patient, caregiver, or healthcare professional, your perspective can contribute to ongoing discussions in this crucial area of medical research.

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