Dataset Information

Cost-Benefit Implication of Gene Expression Profiling and Adjuvant Therapy in Stage IIIA Melanoma.

ABSTRACT:

Background

Indiscriminate use of adjuvant therapy in stage IIIA melanoma is controversial. We sought to model the clinical impact and cost of implementing a gene expression profile (GEP) test to guide adjuvant therapy.

Study design

A Markov decision-analysis model was created to represent resected stage IIIA melanoma with 3 treatment options: observation (OBS), adjuvant pembrolizumab for all patients (ALL), and selective adjuvant therapy (SEL). In the SEL option, only high-risk patients based on GEP stratification were treated with pembrolizumab. Cost of adjuvant therapy was normalized to reflect Medicare reimbursement schedules. The primary outcome was cost per mortality avoided at 10 years.

Results

Model projections for 10-year overall survival were 68% for OBS, 73% for SEL, and 76% for ALL. The estimated incremental cost-per-mortality-avoided (compared to OBS) was $2.1 million for SEL and $2.4 million for ALL. These translate to costs of $583.0K and $697.1K per life-year for the SEL and ALL strategies, respectively.

Conclusions

Routine adjuvant pembrolizumab for stage IIIA melanoma is costly, and risk-stratification by GEP only marginally improves the value of therapy.

SUBMITTER: Hu Y

PROVIDER: S-EPMC7854824 | biostudies-literature | 2020 Nov

REPOSITORIES: biostudies-literature

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Publications

Cost-Benefit Implication of Gene Expression Profiling and Adjuvant Therapy in Stage IIIA Melanoma.

Hu Yinin Y Briggs Andrew A Marchetti Michael A MA Ariyan Charlotte E CE Coit Daniel G DG Bartlett Edmund K EK

Journal of the American College of Surgeons 20200901 5

<h4>Background</h4>Indiscriminate use of adjuvant therapy in stage IIIA melanoma is controversial. We sought to model the clinical impact and cost of implementing a gene expression profile (GEP) test to guide adjuvant therapy.<h4>Study design</h4>A Markov decision-analysis model was created to represent resected stage IIIA melanoma with 3 treatment options: observation (OBS), adjuvant pembrolizumab for all patients (ALL), and selective adjuvant therapy (SEL). In the SEL option, only high-risk pa ...[more]

PMID: 32889093

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Project description:BackgroundOn the basis of data from a phase 2 trial that compared the checkpoint inhibitor ipilimumab at doses of 0.3 mg, 3 mg, and 10 mg per kilogram of body weight in patients with advanced melanoma, this phase 3 trial evaluated ipilimumab at a dose of 10 mg per kilogram in patients who had undergone complete resection of stage III melanoma.MethodsAfter patients had undergone complete resection of stage III cutaneous melanoma, we randomly assigned them to receive ipilimumab at a dose of 10 mg per kilogram (475 patients) or placebo (476) every 3 weeks for four doses, then every 3 months for up to 3 years or until disease recurrence or an unacceptable level of toxic effects occurred. Recurrence-free survival was the primary end point. Secondary end points included overall survival, distant metastasis-free survival, and safety.ResultsAt a median follow-up of 5.3 years, the 5-year rate of recurrence-free survival was 40.8% in the ipilimumab group, as compared with 30.3% in the placebo group (hazard ratio for recurrence or death, 0.76; 95% confidence interval [CI], 0.64 to 0.89; P<0.001). The rate of overall survival at 5 years was 65.4% in the ipilimumab group, as compared with 54.4% in the placebo group (hazard ratio for death, 0.72; 95.1% CI, 0.58 to 0.88; P=0.001). The rate of distant metastasis-free survival at 5 years was 48.3% in the ipilimumab group, as compared with 38.9% in the placebo group (hazard ratio for death or distant metastasis, 0.76; 95.8% CI, 0.64 to 0.92; P=0.002). Adverse events of grade 3 or 4 occurred in 54.1% of the patients in the ipilimumab group and in 26.2% of those in the placebo group. Immune-related adverse events of grade 3 or 4 occurred in 41.6% of the patients in the ipilimumab group and in 2.7% of those in the placebo group. In the ipilimumab group, 5 patients (1.1%) died owing to immune-related adverse events.ConclusionsAs adjuvant therapy for high-risk stage III melanoma, ipilimumab at a dose of 10 mg per kilogram resulted in significantly higher rates of recurrence-free survival, overall survival, and distant metastasis-free survival than placebo. There were more immune-related adverse events with ipilimumab than with placebo. (Funded by Bristol-Myers Squibb; ClinicalTrials.gov number, NCT00636168 , and EudraCT number, 2007-001974-10 .).

Dataset Information

Cost-Benefit Implication of Gene Expression Profiling and Adjuvant Therapy in Stage IIIA Melanoma.

Background

Study design

Results

Conclusions

Publications

Cost-Benefit Implication of Gene Expression Profiling and Adjuvant Therapy in Stage IIIA Melanoma.

Similar Datasets

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