{"title":"具有部分区间缺失数据的混合物验证半参数加速失效时间模型","authors":"Isabel Li, Jun Ma, Benoit Liquet","doi":"10.1002/bimj.202300203","DOIUrl":null,"url":null,"abstract":"<div>\n \n <p>In practical survival analysis, the situation of no event for a patient can arise even after a long period of waiting time, which means a portion of the population may never experience the event of interest. Under this circumstance, one remedy is to adopt a mixture cure Cox model to analyze the survival data. However, if there clearly exhibits an acceleration (or deceleration) factor among their survival times, then an accelerated failure time (AFT) model will be preferred, leading to a mixture cure AFT model. In this paper, we consider a penalized likelihood method to estimate the mixture cure semiparametric AFT models, where the unknown baseline hazard is approximated using Gaussian basis functions. We allow partly interval-censored survival data which can include event times and left-, right-, and interval-censoring times. The penalty function helps to achieve a smooth estimate of the baseline hazard function. We will also provide asymptotic properties to the estimates so that inferences can be made on regression parameters and hazard-related quantities. Simulation studies are conducted to evaluate the model performance, which includes a comparative study with an existing method from the <span>smcure</span> <span>R</span> package. The results show that our proposed penalized likelihood method has acceptable performance in general and produces less bias when faced with the identifiability issue compared to <span>smcure</span>. To illustrate the application of our method, a real case study involving melanoma recurrence is conducted and reported. Our model is implemented in our R package <span>aftQnp</span> which is available from https://github.com/Isabellee4555/aftQnP.</p></div>","PeriodicalId":55360,"journal":{"name":"Biometrical Journal","volume":"66 8","pages":""},"PeriodicalIF":1.3000,"publicationDate":"2024-11-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Mixture Cure Semiparametric Accelerated Failure Time Models With Partly Interval-Censored Data\",\"authors\":\"Isabel Li, Jun Ma, Benoit Liquet\",\"doi\":\"10.1002/bimj.202300203\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div>\\n \\n <p>In practical survival analysis, the situation of no event for a patient can arise even after a long period of waiting time, which means a portion of the population may never experience the event of interest. Under this circumstance, one remedy is to adopt a mixture cure Cox model to analyze the survival data. However, if there clearly exhibits an acceleration (or deceleration) factor among their survival times, then an accelerated failure time (AFT) model will be preferred, leading to a mixture cure AFT model. In this paper, we consider a penalized likelihood method to estimate the mixture cure semiparametric AFT models, where the unknown baseline hazard is approximated using Gaussian basis functions. We allow partly interval-censored survival data which can include event times and left-, right-, and interval-censoring times. The penalty function helps to achieve a smooth estimate of the baseline hazard function. We will also provide asymptotic properties to the estimates so that inferences can be made on regression parameters and hazard-related quantities. Simulation studies are conducted to evaluate the model performance, which includes a comparative study with an existing method from the <span>smcure</span> <span>R</span> package. The results show that our proposed penalized likelihood method has acceptable performance in general and produces less bias when faced with the identifiability issue compared to <span>smcure</span>. To illustrate the application of our method, a real case study involving melanoma recurrence is conducted and reported. Our model is implemented in our R package <span>aftQnp</span> which is available from https://github.com/Isabellee4555/aftQnP.</p></div>\",\"PeriodicalId\":55360,\"journal\":{\"name\":\"Biometrical Journal\",\"volume\":\"66 8\",\"pages\":\"\"},\"PeriodicalIF\":1.3000,\"publicationDate\":\"2024-11-07\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Biometrical Journal\",\"FirstCategoryId\":\"99\",\"ListUrlMain\":\"https://onlinelibrary.wiley.com/doi/10.1002/bimj.202300203\",\"RegionNum\":3,\"RegionCategory\":\"生物学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q4\",\"JCRName\":\"MATHEMATICAL & COMPUTATIONAL BIOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Biometrical Journal","FirstCategoryId":"99","ListUrlMain":"https://onlinelibrary.wiley.com/doi/10.1002/bimj.202300203","RegionNum":3,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"MATHEMATICAL & COMPUTATIONAL BIOLOGY","Score":null,"Total":0}
引用次数: 0
摘要
在实际的生存分析中,即使经过很长一段时间的等待,也可能会出现患者无事件发生的情况,这意味着有一部分人可能永远不会经历感兴趣的事件。在这种情况下,一种补救方法是采用混合治愈考克斯模型来分析生存数据。但是,如果他们的存活时间明显存在加速(或减速)因素,那么加速失效时间(AFT)模型将更受青睐,从而导致混合固化 AFT 模型。在本文中,我们考虑用惩罚似然法估计混合治愈半参数 AFT 模型,其中未知基线危害使用高斯基函数近似。我们允许部分区间校正的生存数据,这些数据可以包括事件时间、左校正时间、右校正时间和区间校正时间。惩罚函数有助于实现基线危害函数的平稳估计。我们还将提供估计值的渐近特性,以便对回归参数和危害相关量进行推断。我们进行了模拟研究来评估模型的性能,其中包括与 smcure R 软件包中现有方法的比较研究。结果表明,我们提出的惩罚似然法总体上具有可接受的性能,与 smcure 相比,在面临可识别性问题时产生的偏差较小。为了说明我们方法的应用,我们进行并报告了一个涉及黑色素瘤复发的真实案例研究。我们的模型在 R 软件包 aftQnp 中实现,该软件包可从 https://github.com/Isabellee4555/aftQnP 获取。
Mixture Cure Semiparametric Accelerated Failure Time Models With Partly Interval-Censored Data
In practical survival analysis, the situation of no event for a patient can arise even after a long period of waiting time, which means a portion of the population may never experience the event of interest. Under this circumstance, one remedy is to adopt a mixture cure Cox model to analyze the survival data. However, if there clearly exhibits an acceleration (or deceleration) factor among their survival times, then an accelerated failure time (AFT) model will be preferred, leading to a mixture cure AFT model. In this paper, we consider a penalized likelihood method to estimate the mixture cure semiparametric AFT models, where the unknown baseline hazard is approximated using Gaussian basis functions. We allow partly interval-censored survival data which can include event times and left-, right-, and interval-censoring times. The penalty function helps to achieve a smooth estimate of the baseline hazard function. We will also provide asymptotic properties to the estimates so that inferences can be made on regression parameters and hazard-related quantities. Simulation studies are conducted to evaluate the model performance, which includes a comparative study with an existing method from the smcureR package. The results show that our proposed penalized likelihood method has acceptable performance in general and produces less bias when faced with the identifiability issue compared to smcure. To illustrate the application of our method, a real case study involving melanoma recurrence is conducted and reported. Our model is implemented in our R package aftQnp which is available from https://github.com/Isabellee4555/aftQnP.
期刊介绍:
Biometrical Journal publishes papers on statistical methods and their applications in life sciences including medicine, environmental sciences and agriculture. Methodological developments should be motivated by an interesting and relevant problem from these areas. Ideally the manuscript should include a description of the problem and a section detailing the application of the new methodology to the problem. Case studies, review articles and letters to the editors are also welcome. Papers containing only extensive mathematical theory are not suitable for publication in Biometrical Journal.