INSPIRED Symposium Part 1: Clinical Variables Associated with Improved Outcomes for Children and Young Adults treated with Chimeric Antigen Receptor T cells for B cell Acute Lymphoblastic Leukemia.
Transplant Cell Ther
; 29(10): 598-607, 2023 Oct.
Article
em En
| MEDLINE
| ID: mdl-37481241
ABSTRACT
Chimeric antigen receptor (CAR) T cell therapy (CAR-T) targeting the CD19 antigen on B cell acute lymphoblastic leukemia (B-ALL) has transitioned from a highly investigational therapy with limited access to a commercial therapy with established toxicities, response and survival rates, and access in numerous countries. With more than a decade of clinical study and 5 years of commercial access, data showing associations with success and failure have emerged. To address functional limitations of CAR-T and overcome constrained sample sizes when studying single-trial or single-center data, collaborative groups, including the Pediatric Real World CAR Consortium, the CAR-Multicenter Analysis, the Center for International Blood and Marrow Transplant Research, and the International BFM Study Group, among others, have been retrospectively interrogating the amassed clinical experience. The high patient numbers and varied clinical experiences compiled by these groups have defined clinical variables impacting CAR-T outcomes. Here we review published CAR-T trials and consortium/collaborative outcomes to establish variables associated with optimal response to CAR-T in children and young adults with B-ALL. We focus on findings with clinical relevance that have emerged, including data implicating pretreatment disease burden, presence of extramedullary disease, nonresponse to prior CD19 antigen targeting (blinatumomab therapy), CAR T cell dose, and fludarabine pharmacokinetics as factors impacting post-CAR-T survival. Additionally, we address the role of collaborative efforts going forward in guiding clinical practice evolution and further optimizing post-CAR-T outcomes.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Leucemia-Linfoma Linfoblástico de Células Precursoras B
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Linfoma de Burkitt
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Leucemia-Linfoma Linfoblástico de Células Precursoras
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Receptores de Antígenos Quiméricos
Tipo de estudo:
Clinical_trials
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Risk_factors_studies
Limite:
Adult
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Child
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Humans
Idioma:
En
Revista:
Transplant Cell Ther
Ano de publicação:
2023
Tipo de documento:
Article