Tisagenlecleucel yields high remission rates among patients with B-cell acute lymphoblastic leukemia (ALL). Yet the drug comes with a significant toxicity profile, according to research published in The New England Journal of Medicine.1

Tisagenlecleucel, a chimeric antigen receptor (CAR)-T cell therapy being investigated for patients with B-cell ALL, showed significant efficacy in previous studies. The drug can provide durable remission, though adverse events (AEs) can be a serious problem with administration. Cytokine release syndrome is a particular concern with CAR-T cell therapy.

For this multi-center phase 2 study (ClinicalTrials.gov Identifier: NCT02435849), researchers evaluated the safety and efficacy among 75 patients with CD19-positive relapsed or refractory B-cell ALL. The primary endpoint was the overall remission rate.

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Among the 75 patients who underwent tisagenlecleucel infusion, the median age was 11 years (range 3-23), the median number of previous therapies was 3 (range, 1-8), and the median marrow blast percentage was 74% (range, 5% to 99%). The median time from enrollment to infusion was 45 days.

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The median follow-up was 13.1 months. The 3-month overall remission rate was 81%; all responders were negative for minimal residual disease. Forty-five patients (60%) had complete remission and 16 patients (21%) had complete remission with incomplete hematologic recovery. The median remission duration was not reached.

The 12-month event-free and overall survival rates were 50% and 76%, respectively.

Ninety-five percent of patients had an AE likely caused by tisagenlecleucel; 73% had a grade 3 or 4 AE likely caused to tisagenlecleucel. Cytokine release syndrome was reported in 77% of patients; nearly half (47%) of all patients were admitted to an ICU for a median of 7 days because of this syndrome.

Neurologic AEs were noted in 10 (13%) patients.

The authors concluded that “tisagenlecleucel produced high remission rates and durable remissions without additional therapy in high-risk pediatric and young adult patients with relapsed or refractory B-cell ALL. The risks associated with tisagenlecleucel are substantial, leading to ICU-level care in some cases, but were mitigated in most patients with supportive measures and cytokine blockade.”


  1. Maude SL, Laetsch TW, Buechner J, et al. Tisagenlecleucel in children and young adults with B-cell lymphoblastic leukemia. N Engl J Med. 2018;378:439-48. doi: 10.1056/NEJMoa1709866