(810) Chimeric Antigen Receptor T Cell (CAR-T) Therapy in Children

Introduction: Chimeric antigen receptor T-cell (CAR-T) therapy in pediatric patients was first approved for the treatment of acute lymphoblastic leukemia (ALL) and large B-cell lymphoma in 2017. Their use has revolutionized the role of immunotherapy in cancer treatment. However, severe toxicities have been described in patients who have received CAR T-Cell therapy.

Methods: We retrospectively analyzed the Kids’ Inpatient database for the year 2019. Hospitalized children from 1 month to 20 years with a procedure of CAR-T therapy were selected using the ICD-10 procedure codes. Univariate was performed to assess the risk of a complication/intervention on mortality. Results are presented as frequency and odds ratio for categorical data and as median with IQR for continuous data.

Results: CAR-T therapy was administered to 198 children who had ALL (79.7%) or Non-Hodgkin’s lymphoma (7.6%). The median age for CAR-T therapy was 12 years (IQR:6-17). Compared to all other discharges CAR-T therapy was common in early adolescents (39.3% vs. 33.0%), males (61.1% vs. 44.1%), Hispanic (38.7% vs. 23.1%), and high-income group (30.0% vs. 18.3%). The complications in patients who received CART therapy included tumor lysis syndrome (TLS) (33.3%), coagulopathy (11.1%), fungal infection (8.1%), viral infection (15.2%), bacterial sepsis (16.2%), seizures (9.6%) and acute kidney injury (13.1%). Interventions included Invasive mechanical ventilation (IMV) (7.6%). The length of stay was 20 days (IQR: 13-35) and charges were $722,144 (IQR: 189,661-1,359,884). Palliative care was documented in 9.6% . Mortality from CAR-T therapy was 6.1%. Mortality risk was higher in children requiring IMV (26.7%, OR=7.9, 95% CI:2.0-30.5), TLS (16.7%, OR=26.2, 95% CI:3.3-207.8). DIC (42.9%, OR=15.0, 95% CI:2.9-77.7), Severe sepsis or septic shock (42.9%, OR=15.0, 95% CI:2.9-77.7), bacterial sepsis (25.0%%, OR=13.4, 95% CI:3.7-47.9), dialysis (50.0%, OR=20.3, 95% CI:3.5-115.2) and ECMO (75%, OR=61.3, 95% CI:5.7-649.5).

Conclusions: The primary indication for CAR-T therapy in children is ALL. The prevalence of morbidity and mortality in children who received CAR-T therapy is high in a national sample.