Health‐Related Quality of Life and Symptom Burden in Pediatric Patients Receiving CAR T‐Cell Therapy: A Prospective, Longitudinal Patient‐Reported Outcomes Study
Angela Steineck, Liyun Zhang, Amy Y. Pan, Mallory R. Taylor, Sara K. Silbert, Haneen Shalabi, Liam Comiskey, Jennifer M. Knight, Lori Wiener, Deena LevineABSTRACT
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.