Tisagenlecleucel Outcomes in R/R Extramedullary ALL

Fabrizio VA, Phillips CL, Lane A, et al. Tisagenlecleucel Outcomes in Relapsed/Refractory Extramedullary ALL: A Pediatric Real World CAR Consortium Report. Blood Advances. 2021; (doi: 10.1182/bloodadvances.2021005564).

Treating relapsed/refractory (R/R) extramedullary (EM) acute lymphoblastic leukemia (ALL) with tisagenlecleucel CD19 CAR T-cells results in similar toxicity, relapse, and survival rates to pediatric bone marrow (BM) disease, according to new research. The Pediatric Real World CAR Consortium retrospectively reviewed 184 infused patients from 15 U.S. institutions. The researchers compared the complete response (CR) rate, overall survival (OS), relapse-free survival (RFS), and duration of B-cell aplasia in patients referred for tisagenlecleucel with both central nervous system disease (CNS3) and non-CNS EM with BM only. This study presents outcomes for 55 patients with EM disease before CAR: 40 with CNS3 and 15 with non-CNS EM. On average, CNS cohort group were 10 years old at infusion and non-CNS EM cohort group were 13 years old. A CR was achieved in 88% (35) of CNS disease patients and 66% (10) of those with non-CNS EM disease. At 24 months, the OS rates in BM and non-BM CNS disease patients were similar to non-CNS EM or BM only cohorts. There was no difference in the 12-month rate of RFS between CNS, non-CNS EM, or BM only patients, and there was no increased toxicity in CNS and non-CNS EM disease. When compared with combined CNS and BM, isolated (iCNS) disease tended to improve OS.

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