ArticleJournal of the National Cancer Institute2019
Cost-Effectiveness of Chimeric Antigen Receptor T-Cell Therapy in Pediatric Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia.
Article in Journal of the National Cancer Institute, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 61 papers, 10 of them syntheses that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
61 citing papers in PubMed, 10 syntheses or guidelines pooled it, 123 citations in OpenAlex.
- Cost-Effectiveness of Chimeric Antigen Receptor (CAR) T-Cell Therapy for Blood Cancers: An Updated Systematic Review.PharmacoEconomics - open · 2026Pooled it
- Patient-level simulation models in cancer care: a systematic review.Frontiers in public health · 2025Pooled it
- Evolving Evidence-Based Value Assessment of One-Time Therapies: Tisagenlecleucel as a Case Study.Applied health economics and health policy · 2024Pooled it
- Capturing the Impact of Constraints on the Cost-Effectiveness of Cell and Gene Therapies: A Systematic Review.PharmacoEconomics · 2023Pooled it
- Dose fractionation of CAR-T cells. A systematic review of clinical outcomes.Journal of experimental & clinical cancer research : CR · 2023Pooled it
- Economic Evidence on Potentially Curative Gene Therapy Products: A Systematic Literature Review.PharmacoEconomics · 2021Pooled it
- Applying State-of-the-Art Survival Extrapolation Techniques to the Evaluation of CAR-T Therapies: Evidence from a Systematic Literature Review.Advances in therapy · 2021Pooled it
- A systematic review of economic evaluations of advanced therapy medicinal products.British journal of clinical pharmacology · 2021Pooled it
- A Systematic Review of the Efforts and Hindrances of Modeling and Simulation of CAR T-cell Therapy.The AAPS journal · 2021Pooled it
- Anti-CD19 CAR-T cell therapy bridge to HSCT decreases the relapse rate and improves the long-term survival of R/R B-ALL patients: a systematic review and meta-analysis.Annals of hematology · 2021Pooled it
- Blinatumomab for treatment of children with acute lymphoblastic leukaemia in Hong Kong: A cost-effectiveness analysis.British journal of haematology · 2026Article
- Challenges and advances in CAR-T cell therapy for B-ALL.Biomarker research · 2026Review
- Review
- The quintessential role for CAR T cell therapy in children, adolescents and young adults with cancer.Nature reviews. Clinical oncology · 2026Review
- Pediatric leukemia: origins, pathogenesis, the role of microenvironment and immunological modulation.Frontiers in immunology · 2026Review
- Posttransplant cells for the win? DLI and adoptive cell therapy to eradicate MRD.Hematology. American Society of Hematology. Education Program · 2025Review
- CAR-T and CAR-NK cell therapies in AML: breaking barriers and charting the future.Journal of translational medicine · 2025Review
- Pharmacoeconomic Profiles of Advanced Therapy Medicinal Products in Rare Diseases: A Systematic Review.Healthcare (Basel, Switzerland) · 2025Review
- Peripheral blood neutrophils contribute to Claudin18.2-specific CAR-T cell treatment resistance in advanced gastric cancer.British journal of cancer · 2025Article
- A Vaccine Against Fibroblast Activation Protein Improves Murine Cardiac Fibrosis by Preventing the Accumulation of Myofibroblasts.Circulation research · 2025Article
1 more citing papers are in PubMed but not listed here.
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Authors and funding
4 authors at 2 institutions in 1 country.
Funding
Abstract
backgroundChimeric antigen receptor T-cell (CAR-T) therapy is a promising new class of cancer therapy but has a high up-front cost. We evaluated the cost-effectiveness of CAR-T therapy among pediatric patients with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL).
methodsWe built a microsimulation model for pediatric patients with relapsed/refractory B-ALL receiving either CAR-T therapy or standard of care. Outcomes included costs, quality of life (health utility), complications, and survival. We measured cost-effectiveness with the incremental cost-effectiveness ratio (ICER), with ICERs under $100 000 per quality-adjusted life-year (QALY) considered cost effective. One-way and probabilistic sensitivity analyses were used to test model uncertainty.
resultsCompared to standard of care, CAR-T therapy increased overall cost by $528 200 and improved effectiveness by 8.18 QALYs, resulting in an ICER of $64 600/QALY. The model was sensitive to assumptions about long-term CAR-T survival, the complete remission rate of CAR-T patients, and the health utility of long-term survivors. The base model assumed a 76.0% one-year survival with CAR-T, although if this decreased to 57.8%, then CAR-T was no longer cost effective. If the complete remission rate of CAR-T recipients decreased from 81% to 56.2%, or if the health utility of disease-free survivors decreased from 0.94 to 0.66, then CAR-T was no longer cost effective. Probabilistic sensitivity analysis found that CAR-T was cost effective in 94.8% of iterations at a willingness to pay of $100 000/QALY.
conclusionCAR-T therapy may represent a cost-effective option for pediatric relapsed/refractory B-ALL, although longer follow-up of CAR-T survivors is required to confirm validity of these findings.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.