Clinical and economic impact of molecular testing for BRAF fusion in pediatric low-grade Glioma
Abstract Background Treatment 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, particu...
| Main Authors: | , , , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
BMC
2022-01-01
|
| Series: | BMC Pediatrics |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s12887-021-03069-1 |
| _version_ | 1818974326309781504 |
|---|---|
| author | Juan David Rios Russanthy Velummailum Julie Bennett Liana Nobre Derek S. Tsang Eric Bouffet Cynthia Hawkins Uri Tabori Avram Denburg Petros Pechlivanoglou |
| author_facet | Juan David Rios Russanthy Velummailum Julie Bennett Liana Nobre Derek S. Tsang Eric Bouffet Cynthia Hawkins Uri Tabori Avram Denburg Petros Pechlivanoglou |
| author_sort | Juan David Rios |
| collection | DOAJ |
| description | Abstract Background Treatment 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. Methods We 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. Results The 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. Conclusions We 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. |
| first_indexed | 2024-12-20T15:38:17Z |
| format | Article |
| id | doaj.art-dbc279a0d15a467f9859386c8c1fd1df |
| institution | Directory Open Access Journal |
| issn | 1471-2431 |
| language | English |
| last_indexed | 2024-12-20T15:38:17Z |
| publishDate | 2022-01-01 |
| publisher | BMC |
| record_format | Article |
| series | BMC Pediatrics |
| spelling | doaj.art-dbc279a0d15a467f9859386c8c1fd1df2022-12-21T19:35:20ZengBMCBMC Pediatrics1471-24312022-01-0122111010.1186/s12887-021-03069-1Clinical and economic impact of molecular testing for BRAF fusion in pediatric low-grade GliomaJuan David Rios0Russanthy Velummailum1Julie Bennett2Liana Nobre3Derek S. Tsang4Eric Bouffet5Cynthia Hawkins6Uri Tabori7Avram Denburg8Petros Pechlivanoglou9Child Health Evaluative Sciences, Peter Gilgan Centre for Research and Learning, The Hospital for Sick ChildrenChild Health Evaluative Sciences, Peter Gilgan Centre for Research and Learning, The Hospital for Sick ChildrenDivision of Haematology/Oncology, The Hospital for Sick ChildrenDivision of Haematology/Oncology, The Hospital for Sick ChildrenDivision of Haematology/Oncology, The Hospital for Sick ChildrenDivision of Haematology/Oncology, The Hospital for Sick ChildrenDepartment of Pathology, Hospital for Sick ChildrenDivision of Haematology/Oncology, The Hospital for Sick ChildrenChild Health Evaluative Sciences, Peter Gilgan Centre for Research and Learning, The Hospital for Sick ChildrenChild Health Evaluative Sciences, Peter Gilgan Centre for Research and Learning, The Hospital for Sick ChildrenAbstract Background Treatment 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. Methods We 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. Results The 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. Conclusions We 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.https://doi.org/10.1186/s12887-021-03069-1Pediatric low-grade GliomaHealth economicsCost-effectivenessHealth technology evaluationPrecision medicineMolecular testing |
| spellingShingle | Juan David Rios Russanthy Velummailum Julie Bennett Liana Nobre Derek S. Tsang Eric Bouffet Cynthia Hawkins Uri Tabori Avram Denburg Petros Pechlivanoglou Clinical and economic impact of molecular testing for BRAF fusion in pediatric low-grade Glioma BMC Pediatrics Pediatric low-grade Glioma Health economics Cost-effectiveness Health technology evaluation Precision medicine Molecular testing |
| title | Clinical and economic impact of molecular testing for BRAF fusion in pediatric low-grade Glioma |
| title_full | Clinical and economic impact of molecular testing for BRAF fusion in pediatric low-grade Glioma |
| title_fullStr | Clinical and economic impact of molecular testing for BRAF fusion in pediatric low-grade Glioma |
| title_full_unstemmed | Clinical and economic impact of molecular testing for BRAF fusion in pediatric low-grade Glioma |
| title_short | Clinical and economic impact of molecular testing for BRAF fusion in pediatric low-grade Glioma |
| title_sort | clinical and economic impact of molecular testing for braf fusion in pediatric low grade glioma |
| topic | Pediatric low-grade Glioma Health economics Cost-effectiveness Health technology evaluation Precision medicine Molecular testing |
| url | https://doi.org/10.1186/s12887-021-03069-1 |
| work_keys_str_mv | AT juandavidrios clinicalandeconomicimpactofmoleculartestingforbraffusioninpediatriclowgradeglioma AT russanthyvelummailum clinicalandeconomicimpactofmoleculartestingforbraffusioninpediatriclowgradeglioma AT juliebennett clinicalandeconomicimpactofmoleculartestingforbraffusioninpediatriclowgradeglioma AT liananobre clinicalandeconomicimpactofmoleculartestingforbraffusioninpediatriclowgradeglioma AT derekstsang clinicalandeconomicimpactofmoleculartestingforbraffusioninpediatriclowgradeglioma AT ericbouffet clinicalandeconomicimpactofmoleculartestingforbraffusioninpediatriclowgradeglioma AT cynthiahawkins clinicalandeconomicimpactofmoleculartestingforbraffusioninpediatriclowgradeglioma AT uritabori clinicalandeconomicimpactofmoleculartestingforbraffusioninpediatriclowgradeglioma AT avramdenburg clinicalandeconomicimpactofmoleculartestingforbraffusioninpediatriclowgradeglioma AT petrospechlivanoglou clinicalandeconomicimpactofmoleculartestingforbraffusioninpediatriclowgradeglioma |