There have been recent successes in the development of potentially curative treatments for pediatric hematologic malignancies. In August 2017, the US Food and Drug Administration (FDA) approved tisagenlecleucel for the treatment of relapsed or refractory B-cell precursor acute lymphoblastic leukemia in patients aged 25 years or younger. Tisagenlecleucel is a chimeric antigen receptor t-cell (CAR-T) therapy, which is a treatment that genetically modifies a patient’s T cells to express a receptor that can bind to the tumor antigen to target and kill cancer cells.1 The safety and efficacy of tisagenlecleucel, as demonstrated in 1 single-arm clinical trial, showed large potential clinical benefit with an overall remission rate of 83% within 3 months of treatment.2 The FDA approval of tisagenlecleucel was historic, as it became the first gene therapy available in the United States.3
Whittington MD, McQueen RB, Campbell JD. Considerations for Cost-effectiveness Analysis of Curative Pediatric Therapies. JAMA Pediatr. 2018;172(5):409–410. doi:10.1001/jamapediatrics.2018.0049
Customize your JAMA Network experience by selecting one or more topics from the list below.
Create a personal account or sign in to: