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

Abstract

e20040 Background: The regulatory approvals in myeloma were predominantly based on the safety and efficacy of the antimyeloma therapies. Health-related quality of life (HRQOL) metrics have not routinely been incorporated into these studies, nor were they a requirement for approval, which explains the dearth of HRQOL studies in myeloma. Recent approvals of chimeric antigen receptor T-cell (CAR-T) therapy targeting the B-cell maturation antigen were no different. While improved clinical outcomes in myeloma led to the FDA and EMA approvals, the CAR-T effects on HRQOL are not well described. Methods: We conducted a cross-sectional observational pilot study to evaluate HRQOL among patients that received CAR-T. Inclusion criteria were age >/= 18 years with a diagnosis of myeloma, had been treated with an autologous CAR-T a minimum of 56 days prior to survey date and had not yet experienced progression as defined by the IMWG criteria. HRQOL measurements were obtained using validated tool, self-administered FACT-MM questionnaire post-CAR-T infusion. The primary endpoint measurement was the FACT-MM composite score. Associations between FACT-MM scores and predictors were analyzed using nonparametric tests. FACT-MM and subscores were analyzed as continuous and dichotomous outcomes. Results: Between December 2021 and July 2022, 22 patients were enrolled (male: 54.55%, white: 54.55%). The median age of patients was 64 (range, 43-86) and the median time to enroll on the study from diagnosis is 6.5 years (3.5-17). Median FACT-MM score was 137.5 (IQR: 18.5) out of a maximum possible score of 164. The median trial outcome index (TOI) which is the sum of the physical and functional well-being (PWB and FWB), a predictor for physical and functional outcomes from treatment (FACIT) was 95 (IQR: 17) out of maximum possible 112. Other descriptive variables of interest were shown in the table. Differences in TOI were found between races (White, African American (AA), & Asian p = 0.04). In our sample, AA and Asian subjects had higher median TOI, therefore race should not be considered to have a negative impact on clinical trial success. Subjects with a history of Gilbert’s syndrome (2 vs 20), reported higher overall FACT-MM scores (p = p=0.04), PWB (p=0.03) and pain-related HRQOL (p = 0.04). Interestingly, we did not find that HRQOL differed by patients that had evidence of CRS or ICANS during CAR-T therapy, or by age, R-ISS, CCI, presence of EMD or prior lines of therapy (LOT). Conclusions: Patients treated with CAR-T have reported high HRQOL metrics at least 2 months out from infusion. Age, RISS, CCI, prior LOT did not impact the HRQOL and may not be the sole criteria used for decision making whether or not to offer CAR-T. [Table: see text]