Tisagenlecleucel in Acute Lymphoblastic Leukemia: A Review of the Literature and Practical Considerations.

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: , and .

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.