The review aimed to characterize the impacts of clinical response, delayed disease progression, and lines of therapy on health-related quality of life (HRQOL) for patients with multiple myeloma.
A literature review published in Clinical Lymphoma, Myeloma, and Leukemia found that delayed disease progression, fewer therapy lines, and deep clinical responses were positively associated with health-related quality of life (HRQOL) for patients with multiple myeloma. The findings support the consideration of treatment response and disease progression as relevant end points in multiple myeloma.
While multiple myeloma treatment has significantly evolved in recent years, it is a heterogenous cancer type that has substantial molecular variation. Additionally, the drugs used to treat multiple myeloma can lead to potentially severe side effects that may significantly impact HRQOL, the authors noted. These include infections, hypertension, steroid-induced diabetes, peripheral neuropathy, and even secondary cancers.
The review aimed to characterize the impact of clinical response and delayed disease progression—both key goals of multiple myeloma therapy in general—on HRQOL from the patient perspective. The impact of line of therapy was also explored.
A total of 41 unique studies were included in the literature review after researchers screened 2198 citations identified in database searches and narrowed it down to 44 records overall. Of those records, 30 included data on HRQOL and disease progression, 5 included data on HRQOL and line of therapy, and 19 included data on HRQOL and treatment response.
The designs and populations of the studies varied, and most HRQOL data came from clinical trials. Real-world data on HRQOL were scarce, the authors noted.
Records relating to disease progression found that progressing disease was linked to decreased patient HRQOL overall. In 3 records from longitudinal studies, patients reported increased QOL from baseline after treatment, followed by a reduction at disease progression.
Few studies reported on the relationship between line of therapy and HRQOL, but most of those studies showed that HRQOL worsened with more lines of therapy.
In the 17 records with data on clinical response and HRQOL, researchers found that a greater depth of response was associated with improved HRQOL. Three records reporting data on minimal residual disease (MRD) showed that patients who tested negative for MRD experienced a longer time to worsened HRQOL compared with those who tested positive for MRD.
The review was limited due to a limited number of studies and the heterogeneity of those studies. There was also minimal real-world evidence identified in the database search.
“This suggests not only a lack of available research in this area, but also the absence of standard reporting methodology,” the authors wrote. “Heterogeneity precluded any ability to conduct a quantitative analysis of the data to support/confirm the findings of this descriptive synthesis.”
Despite the limitations, the findings demonstrate a correlation between progressive disease and worsened HRQOL, as well as a link between increasing lines of therapy and worsened HRQOL. Deeper responses to therapy were correlated with improved HRQOL, on the other hand.
These findings support utilizing delayed progression, fewer lines of therapy, and achieving the deepest possible clinical response as relevant outcomes in multiple myeloma studies. The review also highlights gaps in research that must be addressed, the authors noted.
“To address this gap, HRQOL measures should be collected systematically in an ongoing and standardized manner in future clinical trials and observational studies in patients with MM,” the authors wrote. “Fortunately, a greater number of studies seem to be capturing these data in the form of patient reported outcomes.”
Fonseca R, Tran D, Laidlaw A, et al. Impact of disease progression, line of therapy, and response on health-related quality of life in multiple myeloma: a systematic literature review. Clin Lymphoma Myeloma Leuk. Published online March 17, 2023. doi:10.1016/j.clml.2023.03.005