Treatment of infants with B-cell acute lymphoblastic leukemia (B-ALL) with tisagenlecleucel was effective with acceptable rates of cytokine release syndrome (CRS), according to the results of a registry published in the journal Blood Advances.

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

The retrospective study evaluated data from 14 infants with B-ALL in the Pediatric Real-World CAR Consortium who 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%, with 50% of patients in remission at last follow-up.

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

CRS was common, with any grade occurring among 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.


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