PDF(Pubmed)
Abstract：
BACKGROUND: A recent trial showed that postmenopausal women diagnosed with hormone receptor-positive, human epidermal growth factor receptor-2 (HER2)-negative, lymph node-positive (1-3 nodes) breast cancer with a 21-gene recurrence score of ≤ 25 could safely omit chemotherapy. However, there are limited data on population-level long-term outcomes associated with omitting chemotherapy among diverse women seen in real-world practice.
METHODS: We adapted an established, validated simulation model to generate the joint distributions of population-level characteristics of women diagnosed with early-stage breast cancer in the U.S. Input parameters were derived from cancer registry, meta-analyses, and clinical trial data. The effects of omitting chemotherapy on 10-year distant recurrence-free survival, life-years, and quality adjusted life-years (QALYs) were modeled for premenopausal and postmenopausal women. QALYs were discounted at 3%. Results were evaluated for subgroups stratified by race and ethnicity. Sensitivity analyses included testing results across a range of inputs. The model was validated using the published RxPONDER trial data.
RESULTS: In premenopausal women, the 10-year distant recurrence-free survival rates were 85.3% with chemo-endocrine and 80.1% with endocrine therapy. The estimated life-years and QALYs gained with chemotherapy in premenopausal women were 2.1 and 0.6, respectively. There was no chemotherapy benefit in postmenopausal women. There was no variation in the absolute benefit of chemotherapy across racial or ethnic subgroups. However, there were differences in distant recurrence-free survival rates, life-years, and QALYs across groups. Sensitivity analysis showed similar results. The model closely replicated the RxPONDER trial.
CONCLUSIONS: Modeled population-level outcomes show a small chemotherapy benefit in premenopausal women, but no benefit among postmenopausal women. Simulation modeling provides a useful tool to extend trial data and evaluate population-level outcomes.
METHODS: We adapted an established, validated simulation model to generate the joint distributions of population-level characteristics of women diagnosed with early-stage breast cancer in the U.S. Input parameters were derived from cancer registry, meta-analyses, and clinical trial data. The effects of omitting chemotherapy on 10-year distant recurrence-free survival, life-years, and quality adjusted life-years (QALYs) were modeled for premenopausal and postmenopausal women. QALYs were discounted at 3%. Results were evaluated for subgroups stratified by race and ethnicity. Sensitivity analyses included testing results across a range of inputs. The model was validated using the published RxPONDER trial data.
RESULTS: In premenopausal women, the 10-year distant recurrence-free survival rates were 85.3% with chemo-endocrine and 80.1% with endocrine therapy. The estimated life-years and QALYs gained with chemotherapy in premenopausal women were 2.1 and 0.6, respectively. There was no chemotherapy benefit in postmenopausal women. There was no variation in the absolute benefit of chemotherapy across racial or ethnic subgroups. However, there were differences in distant recurrence-free survival rates, life-years, and QALYs across groups. Sensitivity analysis showed similar results. The model closely replicated the RxPONDER trial.
CONCLUSIONS: Modeled population-level outcomes show a small chemotherapy benefit in premenopausal women, but no benefit among postmenopausal women. Simulation modeling provides a useful tool to extend trial data and evaluate population-level outcomes.
摘要：
背景：最近的一项试验表明，绝经后女性被诊断为激素受体阳性，人表皮生长因子受体2（HER2）阴性，21基因复发评分≤25的淋巴结阳性（1-3个）乳腺癌可以安全地省略化疗。然而,在现实世界的实践中,在不同女性中,与省略化疗相关的人群水平长期结局数据有限.
方法：我们调整了既定的，验证的模拟模型，以生成在美国诊断患有早期乳腺癌的妇女的人口水平特征的联合分布输入参数来自癌症登记，荟萃分析，和临床试验数据。省略化疗对10年无远处复发生存率的影响，生命年,对绝经前和绝经后妇女进行质量调整生命年(QALYs)建模.QALY折扣率为3%。根据种族和种族分层的亚组评估结果。敏感性分析包括一系列输入的测试结果。使用已发布的RxPONDER试验数据验证了该模型。
结果：在绝经前妇女中，化疗-内分泌治疗的10年无远处复发生存率为85.3%,内分泌治疗为80.1%.绝经前妇女化疗后获得的估计生命年和QALYs分别为2.1和0.6。绝经后妇女没有化疗益处。在不同种族或种族亚组之间，化疗的绝对益处没有差异。然而,远处无复发生存率有差异，生命年,和跨群体的QALY。敏感性分析结果相似。该模型紧密复制了RxPONDER试验。
结论：模拟人群水平的结果显示，绝经前妇女的化疗获益较小，但对绝经后妇女没有益处。仿真建模提供了一个有用的工具来扩展试验数据和评估人群水平的结果。
方法：我们调整了既定的，验证的模拟模型，以生成在美国诊断患有早期乳腺癌的妇女的人口水平特征的联合分布输入参数来自癌症登记，荟萃分析，和临床试验数据。省略化疗对10年无远处复发生存率的影响，生命年,对绝经前和绝经后妇女进行质量调整生命年(QALYs)建模.QALY折扣率为3%。根据种族和种族分层的亚组评估结果。敏感性分析包括一系列输入的测试结果。使用已发布的RxPONDER试验数据验证了该模型。
结果：在绝经前妇女中，化疗-内分泌治疗的10年无远处复发生存率为85.3%,内分泌治疗为80.1%.绝经前妇女化疗后获得的估计生命年和QALYs分别为2.1和0.6。绝经后妇女没有化疗益处。在不同种族或种族亚组之间，化疗的绝对益处没有差异。然而,远处无复发生存率有差异，生命年,和跨群体的QALY。敏感性分析结果相似。该模型紧密复制了RxPONDER试验。
结论：模拟人群水平的结果显示，绝经前妇女的化疗获益较小，但对绝经后妇女没有益处。仿真建模提供了一个有用的工具来扩展试验数据和评估人群水平的结果。
