Comparing Survival Extrapolation within All-Cause and Relative Survival Frameworks by Standard Parametric Models and Flexible Parametric Spline Models Using the Swedish Cancer Registry.

IF 3.1 3区 医学 Q2 HEALTH CARE SCIENCES & SERVICES Medical Decision Making Pub Date : 2024-04-01 Epub Date: 2024-02-05 DOI:10.1177/0272989X241227230
Enoch Yi-Tung Chen, Yuliya Leontyeva, Chia-Ni Lin, Jung-Der Wang, Mark S Clements, Paul W Dickman
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Abstract

Background: In health technology assessment, restricted mean survival time and life expectancy are commonly evaluated. Parametric models are typically used for extrapolation. Spline models using a relative survival framework have been shown to estimate life expectancy of cancer patients more reliably; however, more research is needed to assess spline models using an all-cause survival framework and standard parametric models using a relative survival framework.

Aim: To assess survival extrapolation using standard parametric models and spline models within relative survival and all-cause survival frameworks.

Methods: From the Swedish Cancer Registry, we identified patients diagnosed with 5 types of cancer (colon, breast, melanoma, prostate, and chronic myeloid leukemia) between 1981 and 1990 with follow-up until 2020. Patients were categorized into 15 cancer cohorts by cancer and age group (18-59, 60-69, and 70-99 y). We right-censored the follow-up at 2, 3, 5, and 10 y and fitted the parametric models within an all-cause and a relative survival framework to extrapolate to 10 y and lifetime in comparison with the observed Kaplan-Meier survival estimates. All cohorts were modeled with 6 standard parametric models (exponential, Weibull, Gompertz, log-logistic, log-normal, and generalized gamma) and 3 spline models (on hazard, odds, and normal scales).

Results: For predicting 10-y survival, spline models generally performed better than standard parametric models. However, using an all-cause or a relative survival framework did not show any distinct difference. For lifetime survival, extrapolating from a relative survival framework agreed better with the observed survival, particularly using spline models.

Conclusions: For extrapolation to 10 y, we recommend spline models. For extrapolation to lifetime, we suggest extrapolating in a relative survival framework, especially using spline models.

Highlights: For survival extrapolation to 10 y, spline models generally performed better than standard parametric models did. However, using an all-cause or a relative survival framework showed no distinct difference under the same parametric model.Survival extrapolation to lifetime within a relative survival framework agreed well with the observed data, especially using spline models.Extrapolating parametric models within an all-cause survival framework may overestimate survival proportions at lifetime; models for the relative survival approach may underestimate instead.

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利用瑞典癌症登记处,比较标准参数模型和灵活参数样条模型在全因和相对生存框架内的生存率推断。
背景:在健康技术评估中,通常会对限制性平均存活时间和预期寿命进行评估。参数模型通常用于外推。使用相对生存框架的样条模型已被证明能更可靠地估计癌症患者的预期寿命;然而,还需要更多的研究来评估使用全因生存框架的样条模型和使用相对生存框架的标准参数模型:我们从瑞典癌症登记处确定了1981年至1990年期间确诊的5种癌症(结肠癌、乳腺癌、黑色素瘤、前列腺癌和慢性粒细胞白血病)患者,并对其进行随访至2020年。患者按癌症和年龄组(18-59 岁、60-69 岁和 70-99 岁)分为 15 个癌症队列。我们对 2、3、5 和 10 年的随访进行了右删减,并在全因生存和相对生存框架内拟合了参数模型,以便与观察到的 Kaplan-Meier 生存估计值相比,推断出 10 年和终生的生存期。所有队列均采用 6 个标准参数模型(指数模型、Weibull 模型、Gompertz 模型、对数逻辑模型、对数正态模型和广义伽马模型)和 3 个样条模型(危险、几率和正态尺度)进行建模:在预测 10 年生存率方面,样条模型的表现通常优于标准参数模型。然而,使用全因生存或相对生存框架并没有显示出明显的差异。对于终生存活率,从相对存活率框架推断与观察到的存活率吻合得更好,特别是使用样条模型:结论:对于 10 年的外推,我们建议使用样条模型。对于终生存活率的外推,我们建议采用相对存活率框架进行外推,尤其是使用样条模型:对于生存期外推至 10 年,样条线模型的表现通常优于标准参数模型。在全因生存框架内推断参数模型可能会高估终生生存比例,而相对生存方法的模型可能会低估终生生存比例。
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来源期刊
Medical Decision Making
Medical Decision Making 医学-卫生保健
CiteScore
6.50
自引率
5.60%
发文量
146
审稿时长
6-12 weeks
期刊介绍: Medical Decision Making offers rigorous and systematic approaches to decision making that are designed to improve the health and clinical care of individuals and to assist with health care policy development. Using the fundamentals of decision analysis and theory, economic evaluation, and evidence based quality assessment, Medical Decision Making presents both theoretical and practical statistical and modeling techniques and methods from a variety of disciplines.
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