Estimation Of Total Costs In Pediatric And Young Adult Patients With Relapsed Or Refractory Acute Lymphoblastic Leukemia Receiving Tisagenlecleucel From A Us Hospital'S Perspective

INTRODUCTION: Acute lymphoblastic leukemia (ALL) is a hematological malignancy that primarily affects children, adolescents, and young adults. Patients with refractory disease or in second or later relapse have a poor prognosis and are less likely to achieve long-term disease remission. In August 2017, tisagenlecleucel, a Chimeric antigen receptor T-cell (CAR-T) therapy, received approval from the US Food and Drug Administration (FDA) for the treatment of pediatric and young adult patients with B-cell ALL that is refractory or in second or later relapse based on data from the pivotal Phase II trial ELIANA. Since the launch of tisagenlecleucel in ALL, there has been discussion on the expected health resource use (HRU) and costs associated with the treatment beyond the cost of the drug and procedure. The current study aimed to estimate the total costs of tisagenlecleucel for the treatment of pediatric and young adult patients with relapsed or refractory (r/r) ALL from a US hospital's perspective using the HRU and safety data from the ELIANA trial.