Health-Related Quality of Life and Symptom Burden in Pediatric Patients Receiving CAR T-Cell Therapy: A Prospective, Longitudinal Patient-Reported Outcomes Study.

Background

Chimeric antigen receptor (CAR) T-cell therapy has expanded rapidly as an investigational treatment for advanced cancers. Studies in acute lymphoblastic leukemia (ALL) suggest health-related quality of life (HRQOL) may improve by 1-month post-infusion. However, patient-reported outcomes remain insufficiently characterized, particularly across the broader pediatric CAR T-cell population.

Procedure

English- or Spanish-speaking patients (ages 8-25 years) receiving CAR T-cell therapy for any malignancy were recruited from three US pediatric cancer centers. Patient-reported HRQOL (PedsQL) and symptom burden (Memorial Symptom Assessment Scale [MSAS]) were assessed serially through 12 months post-infusion, emphasizing the first month. Linear mixed models characterized score trajectories and examined associations between symptom burden and HRQOL.

Results

Fifty-nine patients completed baseline HRQOL assessments; 41% were treated for ALL. Median age was 17 years and 46% identified as female. Participants completed a median of six assessments, with nearly 75% obtained within the first month. Scores were worst at baseline (mean [SD]: PedsQL Generic: 64.9 [18.4], PedsQL Cancer: 70.0 [16.2], MSAS: 24.4 [19.4]) and improved post-infusion. The greatest gains from baseline to 4 weeks were seen in physical functioning (mean + 8) and worry (mean + 20). Across all timepoints, the most burdensome symptoms were fatigue, worry, pain, nervousness, and anorexia. Symptom burden demonstrated a strong negative correlation with HRQOL (r = - 0.81, p <  0.0001).

Conclusions

Pediatric patients undergoing CAR T-cell therapy experience generally favorable HRQOL trajectories and modest, time-limited symptom burden. Serial patient-reported outcome collection is feasible in this population and provides actionable insight to guide supportive care delivery.