Pediatric Patient Reported Outcomes (PROs) Following Car T Cell Therapy

The use of chimeric antigen receptor-modified T cells (CAR T Cells) has drastically improved cure rates and survival outcomes for pediatric and young adult patients with B lymphoblastic leukemia. While many adverse effects of these types of treatments are well documented through event reporting in clinical trials, more subjective symptoms like fatigue, nausea, and pain are less understood. Systematic collection of this data will enable a better understanding of the treatment experience and may help identify which patients are most at risk of severe side effects following this novel therapy. 

One potential way to collect data on these subjective symptom experiences is through patient reported outcomes (PROs), which has proven to be a feasible, reliable, and valuable strategy. The Ped-PRO-CTCAE, a pediatric-specific PRO measure developed at the behest of the NIH National Cancer Institute (NCI), is used to evaluate symptomatic toxicity in patients receiving chemotherapy. Researchers led by PC3I Faculty Allison Barz Leahy, MD, MSCE are using the Ped-PRO-CTCAE tool, among several other measures, to collect and characterize subjective symptoms that pediatric patients experience following CAR T cell infusion. In addition to subjective symptoms, the study team will also collect data on cognitive function, functional status, health-related quality of life, financial toxicity, and household material hardship. 

In this prospective observational study, patient-reported symptom, toxicity, and quality of life, data will be collected longitudinally for the year following CAR T cell therapy. The results of this study will provide a better understanding of symptom trajectory and duration following CAR T cell therapy and will aid in the generation of toxicity and outcome risk prediction models for this population. While predictive algorithms have been developed for pediatric use, none of them use PRO data, demonstrating the novelty and necessity of this effort. 

Results from this study will be applied in the future to patients and families requiring CAR T cell therapy by contributing to the understanding of the subjective patient experience. The data collected from this study will contribute to the field of predictive modeling, specifically in pediatrics, and be used to inform the development of an early warning system for impending CAR T cell therapy toxicity and relapse. 

ACS Clinician Scientist Development Grant