What question did this study set out to answer?

To assess the clinical efficacy, survival outcomes, and safety profile of tisagenlecleucel in pediatric and young adult patients presenting with relapsed or refractory extramedullary acute lymphoblastic leukemia.

November 18, 2021Blood AdvancesOpen Access

Tisagenlecleucel outcomes in relapsed/refractory extramedullary ALL: a Pediatric Real World CAR Consortium Report

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Authors

VFVanessa A. FabrizioCenter for Cancer and Blood Disorders CPChristine L. PhillipsCincinnati Children's Hospital Medical Center ALAdam LaneCincinnati Children's Hospital Medical Center

Discussion

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Implication

Retrospective cohort study shows robust survival and manageable toxicity with tisagenlecleucel in extramedullary pediatric lymphoblastic leukemia, indicating its efficacy beyond bone marrow.

Key Points

To assess the clinical efficacy, survival outcomes, and safety profile of tisagenlecleucel in pediatric and young adult patients presenting with relapsed or refractory extramedullary acute lymphoblastic leukemia.
Retrospective multicenter study evaluating 184 infused patients across 15 US institutions in the Pediatric Real World CAR Consortium, reporting on 55 patients with pre-infusion extramedullary disease (CNS3, n = 40; non-CNS EM, n = 15).
Compared complete response rates, overall survival, relapse-free survival, duration of B-cell aplasia, and toxicity between extramedullary presentations and bone-marrow-only disease.
Complete response occurred in 88% (35 of 40) of patients with central nervous system involvement versus 66% (10 of 15) of those with non-CNS extramedullary disease.
The 24-month overall survival rates (P = .41) and 12-month relapse-free survival rates (P = .92) demonstrated no significant differences among CNS, non-CNS extramedullary, and bone-marrow-only cohorts.
Treatment-related toxicity showed no significant increase in CNS or non-CNS extramedullary groups compared to bone-marrow-only cases (P = .30), and active CNS disease at infusion did not compromise outcomes.

Cite This Study

Fabrizio et al. (2021) studied this question.

synapsesocial.com/papers/6a1bfd0626cb5670aa9d36e4 https://doi.org/10.1182/bloodadvances.2021005564

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Implication

Retrospective cohort study shows robust survival and manageable toxicity with tisagenlecleucel in extramedullary pediatric lymphoblastic leukemia, indicating its efficacy beyond bone marrow.

Tisagenlecleucel outcomes in relapsed/refractory extramedullary ALL: a Pediatric Real World CAR Consortium Report

Discussion

Member takes

Implication

Key Points

Cite This Study

Tisagenlecleucel outcomes in relapsed/refractory extramedullary ALL: a Pediatric Real World CAR Consortium Report

Discussion

Member takes

Implication

Key Points

Cite This Study

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