Abstract

Cryopreservation of chimeric antigen receptor (CAR) Tcells facilitates shipment, timing of infusions, and storage of subsequent doses. However, reports on the impact of cryopreservation on CAR Tcell efficacy have been mixed. We retrospectively compared clinical outcomes between patients who received cryopreserved versus fresh CAR Tcells for treatment of B cell leukemia across two cohorts of pediatric and young adult patients: those who received anti-CD22 CAR Tcells and those who received bispecific anti-CD19/22 CAR Tcells. Manufacturing methods were consistent within each trial but differed between the two trials, allowing for exploration of cryopreservation within different manufacturing platforms. Among 40 patients who received anti-CD22 CAR Tcells (21 cryopreserved cells and 19 fresh), there were no differences in invivo expansion, persistence, incidence of toxicities, or disease response between groups with cryopreserved and fresh CAR Tcells. Among 19 patients who received anti-CD19/22 CAR Tcells (11 cryopreserved and 8 fresh), patients with cryopreserved cells had similar expansion, toxicity incidence, and disease response, with decreased CAR Tcell persistence. Overall, our data demonstrate efficacy of cryopreserved CAR Tcells as comparable to fresh infusions, supporting cryopreservation, which will be crucial for advancing the field of cell therapy.

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