Health-related quality of life among multiple myeloma patients treated with CAR-T therapy Restricted; Files Only

Gagnon, Samantha (Fall 2022)

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Health-related quality of life among multiple myeloma patients treated with CAR-T therapy 

By Samantha Gagnon

Background: Multiple myeloma (MM) is the second most common hematologic malignancy, with 34,000 new cases diagnosed in the United States annually. There is currently no cure for MM, though treatment options have expanded exponentially in the last few decades, and significantly improved overall survival, with a 5-year relative survival rate of 57.9%. In this context, adoptive cellular therapies have shown to be a feasible and effective treatment option for patients who are refractory to the commonly used antimyeloma agents. Recently, chimeric antigen receptor T-cell (CAR-T) therapy targeting the B-cell maturation antigen (BCMA) has seen improved clinical outcomes (PFS and OS) in treating relapsed/refractory multiple myeloma (RRMM). Positive outcomes and recent Food and Drug Administration (FDA) approval has seen a need for more data on CAR-T effects on health-related quality of life (HRQL). 

Objective: The primary purpose of this pilot study is to investigate predictors of HRQOL measurements among MM patients, without disease progression, who have been treated with CAR-T therapy. Patients and clinicians may utilize the results of this study to make informed treatment decisions and to identify supportive intervention needs for HRQOL improvement in the study population. 

Methods: HRQOL measurements were obtained by study personal using validated tool, self-administered FACT-MM questionnaire post-CAR-T infusion between December 14, 2021, and July 25, 2022. Subjects included in analysis had received CAR-T for treatment of MM a minimum of 56 days prior to survey date and had not yet experienced progression of disease as defined by the International Myeloma Working Group (IMWG) criteria. The primary endpoint measurement was the FACT-MM composite score. Associations between FACT-MM scores and predictors which included demographics, disease and treatment history, and comorbidities were analyzed using nonparametric tests due to left-skewed distribution. FACT-MM and subscores were analyzed as continuous and dichotomous outcomes. 

Results: The study population included twenty-two patients, with a median FACT-MM score of 137.5 (range: 88-156) out of maximum possible 164 points. There was no significant difference in HRQOL measurements between sex, age groups, years since diagnosis, ISS staging, CCI score, or time since infusion. Statistically significant differences in HRQOL measurements including overall FACT-MM, and subscores including myeloma specific, physical well-being and pain side effects were observed between races, certain comorbidities, different therapy class exposures and number of lines of prior therapy.

Conclusion: Patients treated with CAR-T have relatively high levels of HRQOL at least 2 months out from infusion. Significant findings were limited, though due to the extremely small sample size of this pilot study, the results obtained warranted an expansion of the pilot and further study. 

Table of Contents

Table of Contents

Chapter 1: Background and literature review 1

Introduction 1

Epidemiology of multiple myeloma 2

CAR-T therapy for relapsed/refractory multiple myeloma 4

Health-related quality of life 6

Problem statement 8

Purpose 8

Significance 9

Chapter 2: Manuscript 11

Abstract 11

Introduction 12

Methods 13

Results 16

Discussion 19

Conclusion 22

Tables and Figures 24

Chapter 3 42

Future Directions/ Public Health Implications 42

References 47

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