Response Assessment and Post–CAR T-Cell Therapy Management

CD19 chimeric antigen receptor (CAR) T-cell therapy has revolutionized the therapeutic landscape for pediatric and adult patients with relapsed/refractory B-lineage acute lymphoblastic leukemia (ALL) and non-Hodgkin's lymphoma (NHL). Dramatic results, with objective response rates and complete remission rates ranging from 50% to >90%, have been demonstrated across multiple clinical trials resulting in two FDA-approved CAR T-cell products, tisagenlecleucel (Kymriah, Novartis) and axicabtagene ciloleucel (Yescarta, Gilead).1, 2, 3, 4, 5, 6, 7, 8, 9, 10 Long-term, durable responses following CD19 CAR T-cell therapy are variable with 1-year relapse-free survival rates ranging between 51% and 42% and overall survival rates ranging between 78% and 49% in ALL and NHL, respectively.1, 3, 4, 9, 10, 11, 12 Patients receiving CD19 CAR T-cell therapy commonly develop acute side effects including cytokine release syndrome and immune effector cell–associated neurotoxicity syndrome as well as other organ-specific toxicities that require long-term follow-up. In this chapter, we review the outcomes of pediatric and adult patients with relapsed/refractory ALL and NHL following CD19 CAR T-cell therapy and provide organ-focused clinical practice guidelines for evaluations, monitoring, medications, and supportive care measures beyond the first month after CD19 CAR T-cell therapy.