Feasibility of leukapheresis for CAR T-cell production in heavily pre-treated pediatric patients.

ABSTRACT

BACKGROUND: Autologous CD19 chimeric-antigen receptor (CAR) T-cells are an effective salvage therapy for patients with relapsed or refractory B cell malignancies. The essential first step in the production is the collection of mature lymphocytes through leukapheresis. It is a challenging procedure given the fact patients are heavily pretreated and the special considerations of pediatric apheresis. METHODS: We analyzed the data of leukapheresis outcome for CAR T production in a phase 1b/2 clinical trial enrolling 34 children, adolescents and young adults with relapsed or refractory B-cell malignancies. RESULTS: All patients underwent a single leukapheresis. Given a short production time for CAR T-cells, most patients received bridging therapy prior to apheresis. Leukapheresis was performed using peripheral venous access in the majority (82%) of patients, and the remainder required arterial line or central venous access. T-cell collection efficiency (CE) was variable with a median of 18%. No apheresis-related adverse events were noted, and all procedures were successful but two one resulting in lower than target dose (1 × 106 CAR + cells/kg) and the other in failure of CAR T-cell production. CONCLUSIONS: Collection of sufficient T-cells in heavily pretreated pediatric patients via a single apheresis procedure is feasible even with relatively low T-cell CE.