LAUSR

Objective To evaluate the current literature for tisagenlecleucel in the treatment of relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (ALL). Data Sources A literature search of PubMed (inception to June 18, 2020) and ClinicalTrials.gov was conducted using the following search terms: CTL019, chimeric antigen receptor, CAR-T, and tisagenlecleucel. Study Selection and Data Extraction All trials evaluating the use of tisagenlecleucel in B-cell ALL were reviewed and considered for inclusion. Data Synthesis Tisagenlecleucel displayed overall remission rates ranging from 69% to 93% in patients who historically respond extremely poorly to salvage therapy. Remissions were durable, with 12-month relapse-free survival (RFS) rates of 55% to 59%. These promising results are tempered by the unique adverse effect profile of chimeric antigen receptor (CAR) T-cell therapy. Potentially life-threatening cytokine release syndrome (CRS) occurred in 77% to 100% of patients, and immune effector cell–associated neurotoxicity syndrome (ICANS) developed in 31% to 45% of patients receiving tisagenlecleucel. Relevance to Patient Care and Clinical Practice The successful utilization of tisagenlecleucel therapy requires meticulous planning, prudent patient selection, multidisciplinary collaboration, and expert training to ensure optimal patient care. The complex interplay of patient- and treatment-related factors creates problematic barriers that must be expertly navigated by the health care team and authorized treatment center. Conclusions As the first US Food and Drug Administration–approved gene therapy, tisagenlecleucel heralds an immunotherapeutic breakthrough for treating pediatric and young adult patients with r/r B-cell ALL. Many questions surrounding patient-specific gene and cellular therapies remain, but their transformative potential in cancer care remains promising.