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

Myers, Regina M.; Jacoby, Elad; Pulsipher, Michael A.; Pasquini, Marcelo C.; Grupp, Stephan A.; Shah, Nirali N.; Laetsch, Theodore W.; Curran, Kevin J.; Schultz, Liora

doi:10.1016/j.jtct.2023.07.016

Cited by 5 publications

(2 citation statements)

References 57 publications

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“…CAR-T cell therapy has demonstrated remarkable success in treating pediatric refractory/relapsed B-ALL, significantly improving outcomes for children who may not have responded well to conventional treatments [47]. Infants were initially excluded from studies involving CAR-T cell therapy due to concerns about the heightened risk of toxicity in this age group.…”

Section: Car-t Cellsmentioning

confidence: 99%

Infant Acute Lymphoblastic Leukemia—New Therapeutic Opportunities

Kulczycka,

Derlatka,

Tasior

et al. 2024

IJMS

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Infant acute lymphoblastic leukemia (Infant ALL) is a kind of pediatric ALL, diagnosed in children under 1 year of age and accounts for less than 5% of pediatric ALL. In the infant ALL group, two subtypes can be distinguished: KMT2A-rearranged ALL, known as a more difficult to cure form and KMT2A- non-rearranged ALL with better survival outcomes. As infants with ALL have lesser treatment outcomes compared to older children, it is pivotal to provide novel treatment approaches. Progress in the development of molecularly targeted therapies and immunotherapy presents exciting opportunities for potential improvement. This comprehensive review synthesizes the current literature on the epidemiology, clinical presentation, molecular genetics, and therapeutic approaches specific to ALL in the infant population.

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Section: Car-t Cellsmentioning

confidence: 99%

Infant Acute Lymphoblastic Leukemia—New Therapeutic Opportunities

Kulczycka,

Derlatka,

Tasior

et al. 2024

IJMS

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“…The ELIANA study demonstrated OS of 63% and event-free survival (EFS) of 44% three years following tisagenlecleucel CAR T-cell therapy [ 4 ]. Since regulatory approval, a growing number of real-world cohorts have reported results generally consistent with those of ELIANA [ 5 ]. However, the EFS definition utilised in ELIANA did not include all events such as early B-cell recovery or emergence of measurable residual disease (MRD), both of which may require further intervention.…”

Section: Introductionmentioning

confidence: 95%

Intention-to-treat outcomes utilising a stringent event definition in children and young people treated with tisagenlecleucel for r/r ALL through a national access scheme

Oporto Espuelas,

Burridge,

Kirkwood

et al. 2024

Blood Cancer J.

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CAR T-cell therapy has transformed relapsed/refractory (r/r) B-cell precursor acute lymphoblastic leukaemia (B-ALL) management and outcomes, but following CAR T infusion, interventions are often needed. In a UK multicentre study, we retrospectively evaluated tisagenlecleucel outcomes in all eligible patients, analysing overall survival (OS) and event-free survival (EFS) with standard and stringent definitions, the latter including measurable residual disease (MRD) emergence and further anti-leukaemic therapy. Both intention-to-treat and infused cohorts were considered. We collected data on feasibility of delivery, manufacture, toxicity, cause of therapy failure and followed patients until death from any cause. Of 142 eligible patients, 125 received tisagenlecleucel, 115/125 (92%) achieved complete remission (CR/CRi). Severe cytokine release syndrome and neurotoxicity occurred in 16/123 (13%) and 10/123 (8.1%), procedural mortality was 3/126 (2.4%). The 2-year intent to treat OS and EFS were 65.2% (95%CI 57.2–74.2%) and 46.5% (95%CI 37.6–57.6%), 2-year intent to treat stringent EFS was 35.6% (95%CI 28.1–44.9%). Median OS was not reached. Sixty-two responding patients experienced CAR T failure by the stringent event definition. Post failure, 1-year OS and standard EFS were 61.2% (95%CI 49.3–75.8) and 55.3% (95%CI 43.6–70.2). Investigation of CAR T-cell therapy for B-ALL delivered on a country-wide basis, including following patients beyond therapy failure, provides clinicians with robust outcome measures. Previously, outcomes post CAR T-cell therapy failure were under-reported. Our data show that patients can be successfully salvaged in this context with good short-term survival.

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Evolving strategies for addressing CAR T-cell toxicities

Rankin,

Duncan,

Allen

et al. 2024

Cancer Metastasis Rev

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The field of chimeric antigen receptor (CAR) T-cell therapy has grown from a fully experimental concept to now boasting a multitude of treatments including six FDA-approved products targeting various hematologic malignancies. Yet, along with their efficacy, these therapies come with side effects requiring timely and thoughtful interventions. In this review, we discuss the most common toxicities associated with CAR T-cells to date, highlighting risk factors, prognostication, implications for critical care management, patient experience optimization, and ongoing work in the field of toxicity mitigation. Understanding the current state of the field and standards of practice is critical in order to improve and manage potential toxicities of both current and novel CAR T-cell therapies as they are applied in the clinic.

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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

Cited by 5 publications

References 57 publications

Infant Acute Lymphoblastic Leukemia—New Therapeutic Opportunities

Infant Acute Lymphoblastic Leukemia—New Therapeutic Opportunities

Intention-to-treat outcomes utilising a stringent event definition in children and young people treated with tisagenlecleucel for r/r ALL through a national access scheme

Evolving strategies for addressing CAR T-cell toxicities

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