Financial toxicity and association with treatment outcomes during pediatric CAR T therapy.

Abstract

10028 Background: Chimeric antigen receptor T cell (CAR) therapy has revolutionized the treatment of relapsed/refractory pediatric B cell malignancies. However, the financial toxicity (FT) experienced by families is unknown despite the likely high burden imposed by clinic visits, hospital admissions, and potential travel/relocation to referral centers. We present the first report of pediatric FT among families of children receiving CAR therapy including prevalence, trajectory, and impact on outcomes. Methods: This is a prospective cohort analysis of patient (pt) and caregiver-reported FT outcomes for pediatric and young adult pts receiving CD19-directed CAR therapy at the Children’s Hospital of Philadelphia. Caregivers (for pts < 18 years old) or pts (if ≥ 18 years old) completed the Comprehensive Score for Financial Toxicity (COST) prior to CAR infusion (baseline), 1 month, and 3 months post-infusion. COST is a self-report FT measure that has been used in adults with specific validated grading criteria (0-3) anchored on independent differences in health-related quality of life. We described baseline FT and tested its association with treatment-related toxicity (occurrence of any cytokine release syndrome [CRS]) and outcomes (complete response [CR] at day 28) using multivariable models adjusted for time from diagnosis, prior CAR exposure, and disease burden at infusion. FT was hypothesized to impact outcomes through physiologic stress affecting immunologic response. In pts with COST data through 3 months, we described longitudinal FT trajectories. Results: From 9/2019-12/2024, 144 pts (33% racial/ethnic minority, 8% public insurance) or their caregivers (28% less than college degree, 22% annual household income < $50,000) completed baseline COST measures. 94% of the cohort had caregiver-reported FT. 81% of pts were external referrals. The median baseline COST score was 20.0 (IQR: 11.0-29.0), which corresponds to Grade 1 FT. Two-thirds of pts reported FT, with 37%, 29%, and < 1% of families experiencing grades 1-3 FT, respectively. 80% of pts had CRS; 89% had CR at day 28. Baseline FT was not associated with CRS or day 28 response (all p > 0.2). A subset of n = 80 had complete longitudinal data and follow up of at least 3 months. In this cohort, median COST score at baseline, month 1, and month 3 was 19.5 (IQR: 10.0-29.3), 18.5 (IQR: 11.0-27.3), 16.0 (IQR: 9.8-27.0), respectively. Strikingly, 23% and 25% of pts had clinically worse grades of FT at 1 month and 3 months post-infusion (compared to baseline), respectively. Race/ethnicity, language, insurance, caregiver education, and income were not associated with worsened FT over time. Conclusions: There is a high prevalence of FT at time of CAR infusion for families of pediatric and young adult pts. Reassuringly, FT at the time of infusion was not associated with two key treatment outcomes, but more investigation is needed. For a notable proportion of families, FT worsens over time and highlights the need for interventions to address cumulative financial burden.