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PLASTIC RECONSTRUCTIVE AND REGENERATIVE SURGERY

Review

Vol. 23 No. 5 (2025): Blood Transfusion 5-2025 (September-October)

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

Authors

Alex Moretti , Chiara Buracchi , Sara Napolitano , Valentina Baldini , Giuseppe Gaipa , Andrea Biondi , Adriana Cristina Balduzzi

Key words: Chimeric antigen receptor, acute lymphoblastic leukemia, adoptive immunotherapy, pediatric
Doi: 10.2450/BloodTransfus.952
Cellular therapy and regenerative medicine
Publication Date: 2025-04-16

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. 

References

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Authors

Alex Moretti Tettamanti Center, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy

Chiara Buracchi Pediatric Transplant Unit, Fondazione IRCCS San Gerardo Dei Tintori, Monza, Italy

Sara Napolitano Pediatric Transplant Unit, Fondazione IRCCS San Gerardo Dei Tintori, Monza, Italy

Valentina Baldini Blood Transfusion Center, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy

Giuseppe Gaipa Tettamanti Center, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy

Andrea Biondi Tettamanti Center, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy

Adriana Cristina Balduzzi Pediatric Transplant Unit, Fondazione IRCCS San Gerardo Dei Tintori, Monza, Italy

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