Patient-reported quality of life and symptoms in children treated with chimeric antigen receptor (CAR) T cell therapy: A prospective cohort study.

e22021 Background: Chimeric Antigen Receptor (CAR) T cell therapy is a standard therapy for relapsed Acute Lymphoblastic Leukemia (ALL) and remains an important investigative therapy in other pediatric malignancies. Current understanding of the patient experience with this therapy is limited to trends in health-related quality of life (HRQOL) in patients with leukemia without understanding underlying contributing factors, such as symptom burden. This study aims to describe HRQOL and symptoms in children treated with CAR T cell therapy. Methods: English or Spanish-speaking patients (8-25 years) undergoing CAR T cell therapy (any malignancy) were recruited from three pediatric cancer centers. Patient and parent proxy participants completed serial measures of HRQOL (PedsQL) and symptom burden (MSAS) for one year post-infusion (baseline, weekly until +4 weeks, monthly until +3 months, quarterly until +12 months; total 10 timepoints). Summary statistics and differences of time using least square means were calculated for each scale and timepoint, adjusted for clinical and demographic covariates. Results: N=58 patients and n=65 parents completed baseline assessments, including 41 patient/parent dyads. Five patients (8%) and nine parents (14%) remained on study at 12-months. Withdrawal from study was due to death (24%), loss to follow up (22%), subsequent transplant (16%), active withdrawal (7%), or other (10%). Patient median age was 16 years (range: 8-25); 77% identified as white, 51% identified as female. Nearly half were treated for ALL, 30% for sarcoma, and 14% for a central nervous system tumor. Parent reporters were predominantly biological mothers (77%) with median age 44 years. Table 1 shows scores, sorted by reporter, and most burdensome symptoms by timepoint. Time was the only significant covariate in longitudinal score difference. Conclusions: HRQOL and symptom burden improve early and significantly following CAR T cell therapy, regardless of patient or proxy report. Patient reported outcome (PRO) scores. Measure Reporter Timepoint (median [IQR]) Baseline +1 w +4 w +6 mo +12 mo PedsQL Generic Patient 69.0 [53.3-78.3] 66.3 [52.2-81.5] 78.8 [68.5-92.4] 80.4 [75-89.1] 90.2 [86.9-92.4] Parent 61.4 [50-73.4] 60.9 [45.6-72.8] 71.7 [53.8-91.3] 69.6 [55.9-83.7] 57.6 [54.3-79.9] PedsQL Cancer Patient 72.2 [57.9-81.5] 75.5 [63.4-83.3] 84.3 [73.1-92.6] 87.9 [74.1-92.6] 83.3 [74.1-86.1] Parent 63.9 [57.4-76.8] 70.4 [59.3-80.6] 82.9 [67.6-89.8] 74.1 [61.1-85.2] 74.5 [58.5-93.5] MSAS Patient 18.9 [9.3-33.0] 13.7 [6.2-29.6] 2.6 [0-12.9] 0 [0-5.5] 5.5 [0-13.4] Parent 11.9 [5.6-27.5] 13.9 [5.6-23.1] 4.0 [0-11.1] 8.8 [0-22.9] 5.2 [0-9.9] Most burdensome symptoms Fatigue, pain, anorexia Fatigue, pain, worry Fatigue, insomnia, worry Fatigue, pain, nausea Anxiety, sad, concentration