Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia.

ABSTRACT:

Background

In a single-center phase 1-2a study, the anti-CD19 chimeric antigen receptor (CAR) T-cell therapy tisagenlecleucel produced high rates of complete remission and was associated with serious but mainly reversible toxic effects in children and young adults with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL).

Methods

We conducted a phase 2, single-cohort, 25-center, global study of tisagenlecleucel in pediatric and young adult patients with CD19+ relapsed or refractory B-cell ALL. The primary end point was the overall remission rate (the rate of complete remission or complete remission with incomplete hematologic recovery) within 3 months.

Results

For this planned analysis, 75 patients received an infusion of tisagenlecleucel and could be eval

SUBMITTER: Maude SL 

PROVIDER: S-EPMC5996391 | biostudies-literature | 2018 Feb

REPOSITORIES: biostudies-literature