Clinical and economic impact of molecular testing for BRAF fusion in pediatric low-grade Glioma

BMC Pediatrics

Russanthy Velummailum¹  Juan David Rios¹  Avram Denburg²  Petros Pechlivanoglou³  Cynthia Hawkins⁴  Liana Nobre⁵  Uri Tabori⁵  Julie Bennett⁵  Eric Bouffet⁵  Derek S. Tsang⁶ 

[1] Child Health Evaluative Sciences, Peter Gilgan Centre for Research and Learning, The Hospital for Sick Children, 686 Bay Street, 11th Floor – L4 East, M5G 0A4, Toronto, ON, Canada;Child Health Evaluative Sciences, Peter Gilgan Centre for Research and Learning, The Hospital for Sick Children, 686 Bay Street, 11th Floor – L4 East, M5G 0A4, Toronto, ON, Canada;Division of Haematology/Oncology, The Hospital for Sick Children, Toronto, Canada;Institute of Health Policy, Management and Evaluation, Dalla Lana School of Public Health, University of Toronto, Toronto, Canada;Child Health Evaluative Sciences, Peter Gilgan Centre for Research and Learning, The Hospital for Sick Children, 686 Bay Street, 11th Floor – L4 East, M5G 0A4, Toronto, ON, Canada;Institute of Health Policy, Management and Evaluation, Dalla Lana School of Public Health, University of Toronto, Toronto, Canada;Department of Pathology, Hospital for Sick Children, Toronto, ON, Canada;Division of Haematology/Oncology, The Hospital for Sick Children, Toronto, Canada;Division of Haematology/Oncology, The Hospital for Sick Children, Toronto, Canada;Radiation Medicine Program, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada;

关键词: Pediatric low-grade Glioma;    Health economics;    Cost-effectiveness;    Health technology evaluation;    Precision medicine;    Molecular testing;    Pediatric oncology;   

DOI  :  10.1186/s12887-021-03069-1

来源: Springer

【 摘 要 】

BackgroundTreatment personalization via tumor molecular testing holds promise for improving outcomes for patients with pediatric low-grade glioma (PLGG). We evaluate the health economic impact of employing tumor molecular testing to guide treatment for patients diagnosed with PLGG, particularly the avoidance of radiation therapy (RT) for patients with BRAF-fusion.MethodsWe performed a model-based cost-utility analysis comparing two strategies: molecular testing to determine BRAF fusion status at diagnosis against no molecular testing. We developed a microsimulation to model the lifetime health and cost outcomes (in quality-adjusted life years (QALYs) and 2018 CAD, respectively) for a simulated cohort of 100,000 patients newly diagnosed with PLGG after their initial surgery.ResultsThe life expectancy after diagnosis for individuals who did not receive molecular testing was 39.01 (95% Confidence Intervals (CI): 32.94;44.38) years and 40.08 (95% CI: 33.19;45.76) years for those who received testing. Our findings indicate that patients who received molecular testing at diagnosis experienced a 0.38 (95% CI: 0.08;0.77) gain in QALYs and $1384 (95% CI: $-3486; $1204) reduction in costs over their lifetime. Cost and QALY benefits were driven primarily by the avoidance of long-term adverse events (stroke, secondary neoplasms) associated with unnecessary use of radiation.ConclusionsWe demonstrate the clinical benefit and cost-effectiveness of molecular testing in guiding the decision to provide RT in PLGG. While our results do not consider the impact of targeted therapies, this work is an example of the value of simulation modeling in assessing the long-term costs and benefits of precision oncology interventions for childhood cancer, which can aid decision-making about health system reimbursement.

【 授权许可】

CC BY