Tisagenlecleucel is effective and produces acceptable rates of cytokine release syndrome (CRS) in infants with B-cell acute lymphoblastic leukemia (B-ALL), according to a study published in Blood Advances.

Tisagenlecleucel is already approved by the US Food and Drug Administration for the treatment of relapsed/refractory B-ALL in younger patients, but patients younger than 3 years of age were excluded from trials supporting approval. The aim of the current study was to evaluate the outcomes of patients in this age group who received tisagenlecleucel.

Researchers evaluated data from 14 infants with B-ALL in the Pediatric Real-World CAR Consortium. The patients received tisagenlecleucel between 2017 and 2020. The median follow-up was 231 days.


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The majority of patients achieved minimal residual disease–negative remission, at 64%, and 50% of patients were still in remission at last follow-up.

There were 5 patients (35.7%) who were refractory to tisagenlecleucel, all of whom harbored high disease burden at the time of tisagenlecleucel infusion. Of these patients, 3 died of progressive disease, and 1 died of transplant-related mortality.

CRS was common, with any grade occurring in 79% of patients. There were 3 patients who developed grade 3-4 CRS, 2 of whom required immunosuppression. There were no reports of neurotoxicity.

The authors concluded that “real-world use of tisagenlecleucel in infant B-ALL shows that this novel therapy can be effective and safe in a highly aggressive leukemia.”

Disclosures: Some of the study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of disclosures.

Reference

Moskop A, Pommert L, Baggott C, et al. Real-world use of tisagenlecleucel in infant acute lymphoblastic leukemia. Blood Adv. 2022;6:4251-4255. doi:10.1182/bloodadvances.2021006393

This article originally appeared on Hematology Advisor