Management guidelines for paediatric patients receiving chimeric antigen receptor T cell therapy.

Mahadeo KM1, Khazal SJ2, Abdel-Azim H3, Fitzgerald JC4, Taraseviciute A5, Bollard CM6, Tewari P7, Duncan C8, Traube C9, McCall D2, Steiner ME10, Cheifetz IM11, Lehmann LE8, Mejia R12, Slopis JM13, Bajwa R14, Kebriaei P15, Martin PL16, Moffet J16, McArthur J17, Petropoulos D2, O'Hanlon Curry J2, Featherston S2, Foglesong J2, Shoberu B18, Gulbis A19, Mireles ME19, Hafemeister L2, Nguyen C2, Kapoor N3, Rezvani K15, Neelapu SS20, Shpall EJ15; Pediatric Acute Lung Injury and Sepsis Investigators (PALISI) Network.

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Abstract

In 2017, an autologous chimeric antigen receptor (CAR) T cell therapy indicated for children and young adults with relapsed and/or refractory CD19+ acute lymphoblastic leukaemia became the first gene therapy to be approved in the USA. This innovative form of cellular immunotherapy has been associated with remarkable response rates but is also associated with unique and often severe toxicities, which can lead to rapid cardiorespiratory and/or neurological deterioration. Multidisciplinary medical vigilance and the requisite health-care infrastructure are imperative to ensuring optimal patient outcomes, especially as these therapies transition from research protocols to standard care. Herein, authors representing the Pediatric Acute Lung Injury and Sepsis Investigators (PALISI) Network Hematopoietic Stem Cell Transplantation (HSCT) Subgroup and the MD Anderson Cancer Center CAR T Cell Therapy-Associated Toxicity (CARTOX) Program have collaborated to provide comprehensive consensus guidelines on the care of children receiving CAR T cell therapy.