Symptom Burden and Health-Related Quality of Life in Adults with Multiple Myeloma

AbstractProblem and purpose Multiple myeloma (MM), a cancer of the plasma cells, is the second most common hematologic malignancy in the United States with an estimated 34,920 new cases and 12,410 deaths in 2021. Though still considered incurable, treatment advances in the last 20 years have dramatically extended survival for many with MM. Overall median survival has increased from approximately two years in 2000 to more than five years in 2020. Though survival has improved, research suggests that those with MM experience high symptom burden and poor health related quality of life (HRQoL), though these outcomes are not well described in the literature. The purpose of this dissertation is to explore symptom burden and HRQoL outcomes in adults with MM. Methods The aims of this dissertation were achieved through: (1) a systematic review of symptoms and HRQoL in adults with relapsed/refractory multiple myeloma (RRMM); (2) a qualitative descriptive study of patients’ experiences living with MM; and (3) a cross-sectional observational survey study exploring symptoms and HRQOL among adults with MM recruited through the Duke Cancer Institute (DCI) Tumor Registry. Our systematic review of the literature aimed to summarize what is known about symptoms and HRQoL in adults with RRMM and assess patient-reported outcome (PRO) reporting quality. Next, our qualitative study explored the physical, emotional, and social impacts of MM and its treatment through semi-structured interviews of MM patients and clinicians. Results from the systematic review and qualitative study informed the design of a cross-sectional observational study of symptom burden and HRQoL in adults with MM. Those with MM diagnosed between the years 2000 to 2019 were identified through the DCI and mailed surveys assessing symptom burden and HRQoL. Results Our systematic review included 30 manuscripts, 25 of which were clinical trial reports. Pain, fatigue, and emotional function were the most commonly reported PROs. Prevalence and severity of symptoms and HRQoL outcomes were rarely reported, limiting our ability to summarize symptom burden and HRQoL. Further, our evaluation of PRO reporting quality revealed significant reporting deficiencies. Findings from our qualitative study highlighted the serious, unrelenting, and far-reaching impacts of MM and the ongoing nature of its treatment. Four themes emerged from our analysis of interviews with 15 MM patients and 10 MM clinicians: 1) treatment without end; 2) social impacts and isolation; 3) ongoing financial pressure; and 4) relationship impacts. In our cross-sectional study among adults with MM, 690 completed surveys were returned for a response rate of 64.7%. The most commonly reported moderate to severe symptoms were pain (47.6%), fatigue (44.3%), insomnia (32%), decreased libido (31%), and peripheral neuropathy (30.0%). More than half (55.7%) of MM patients reported three or more moderate to severe symptoms. Younger age, more comorbidities, and not being in remission were independently associated with increased symptom burden. MM patients experienced significantly worse physical health, mental health and physical function when compared to the general population (p<.0001), though social function did not differ significantly (p = 0.09). Lower socioeconomic status, lesser quality of social support, more comorbidities, past radiation therapy, and not being in remission were significantly associated with worse HRQoL outcomes among patients with MM. Conclusion Our systematic review revealed that the existing MM literature provides limited evidence of the prevalence or severity of symptoms and HRQoL challenges of patients with MM and is further hindered by suboptimal reporting quality. Our qualitative study revealed that the far-reaching negative impacts of MM on HRQOL are experienced in the context of unrelenting treatment. Our survey-based study revealed an alarmingly high symptom burden and poor HRQoL among MM patients. Given this, MM patients would greatly benefit from a concerted and multidisciplinary effort to identify and address their many needs. Future research is needed to better describe complex MM treatment patterns and investigate strategies to better meet the needs of patients with MM.