Chimeric antigen receptor therapy for hematological malignancies: a pediatric perspective from leukapheresis to infusion

Abstract

Immunotherapy, particularly chimeric antigen receptor T cells (CAR T)s, has changed the landscape of B-cell malignancy treatment and represents a promising approach to cancer therapy. The use of CAR T-based therapy in pediatric patients presents several critical issues such as the quality of the leukapheresis process and the treatment-related toxicity. Nevertheless, the experience with anti-CD19 CAR Ts in treating pediatric B-cell precursor acute lymphoblastic leukemia (BCP-ALL) demonstrated its feasibility and efficacy with a complete response rate greater than 80%. Although CAR T therapy is still in its infancy, the growing clinical experience and expanding body of literature are gradually enhancing the management of complications and patient monitoring. Yet, in vivo, CAR T persistence issues emerged and highlighted the need for continuous investigations to improve the long-term efficacy of CAR T cell therapy. Concurrently, it is crucial to expand the use of CAR T cells in the treatment of pediatric tumors other than BCP-ALL and to extend access to such therapy. In this review, we outline the journey of treating pediatric patients with CAR T products, covering the process from referral to long-term monitoring, while also addressing key concerns and future perspectives.