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作者(中文):戴子翔
作者(外文):Dai, Tzu-Hsiang
論文名稱(中文):倖存資料之穩健因果最佳治療估計
論文名稱(外文):Robust Causal Estimation of Optimal Treatment Regime with Survival Data
指導教授(中文):鄭又仁
指導教授(外文):Cheng, Yu-Jen
口試委員(中文):謝文萍
黃冠華
口試委員(外文):Hsieh, Wen-Ping
Huang, Guan-Hua
學位類別:碩士
校院名稱:國立清華大學
系所名稱:統計學研究所
學號:107024522
出版年(民國):109
畢業學年度:108
語文別:英文
論文頁數:98
中文關鍵詞:倖存分析
外文關鍵詞:survival analysis
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決定治療的準則(a treatment regime) 是根據每個病患的身體狀況去指派個別的治療方法。在倖存資料中,常會存在著選擇偏誤以及自變數在接受不同治療的兩群分佈不平均的問題。因此我們會使用治療機率倒數加權的方法以及Rubin因果模型來修正,否則結果不具一致性(consistent)也沒有因果推論(causal inference)的解釋。本論文主要研究利用修正後治療機率倒數加權(IPSW)以及具雙穩健性的方法(AIPSW)去估計最佳治療的準則,使得整體病人或是接受特定治療的病人在此準則下接受的治療可以達到限制平均生存時間(RMST)最大。我們提出的方法會以模擬驗證並應用在真實資料中。
A treatment regime is the way of assigning treatment to each patients according to their own characteristics. In our survival data, there might exist both selection bias and imbalanced covariates between different treatments. The inverse probability weighting method and Rubin causal model are used to adjust those biases. Besides, these methods not only provide the consistency to estimators but also make the causal inference available. In this thesis, we propose both Inverse propensity score weighted (IPSW) and augmented Inverse propensity score weighted (AIPSW) to estimate the optimal treatment regime that maximizes the restricted mean survival time (RMST) for all patients and those who received the specified treatment. Finally, we evaluate the proposed methods by the simulation studies and also applied those methods to the real data.
1 Introduction 1
2 Literature Review 3
2.1 Survival Analysis . . . . . . . . . . . . . . . . . . . . . . . . . . . . 3
2.1.1 Kaplan-Meier Estimator . . . . . . . . . . . . . . . . . . . . 3
2.1.2 Cox Propotional Hazard Regression model . . . . . . . . . . 4
2.2 Selection Bias . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 4
2.3 Causal Inference . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 4
2.3.1 Propensity score . . . . . . . . . . . . . . . . . . . . . . . . 5
2.3.2 Assumptions . . . . . . . . . . . . . . . . . . . . . . . . . . . 5
2.3.3 Causal survival function . . . . . . . . . . . . . . . . . . . . 6
2.3.4 Average Treatment Effect (ATE) . . . . . . . . . . . . . . . 6
2.3.5 Average Treatment Effect on the Treated (ATT) . . . . . . . 7
3 Model and Methodology 7
3.1 Notation . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 7
3.2 Estimation Procedure . . . . . . . . . . . . . . . . . . . . . . . . . . 8
3.2.1 Propensity score . . . . . . . . . . . . . . . . . . . . . . . . 8
3.2.2 Balancing weights . . . . . . . . . . . . . . . . . . . . . . . . 9
3.2.3 Inverse propensity score weighted estimators . . . . . . . . . 10
3.2.4 Augmented inverse propensity score weighted estimators . . 11
3.2.5 Computational Aspects . . . . . . . . . . . . . . . . . . . . . 14
3.2.6 Restricted mean survival . . . . . . . . . . . . . . . . . . . . 14
4 Asymptotic Properties 15
4.1 Inverse propensity score weighted estimators . . . . . . . . . . . . . 15
4.2 Augmented inverse propensity score weighted estimators . . . . . . 16
5 Simulation Studies 17
5.1 Data Setting . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17
5.2 Results . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 18
5.2.1 Compare Different Scenarios . . . . . . . . . . . . . . . . . . 18
5.2.2 Compare Different Estimators . . . . . . . . . . . . . . . . . 20
6 Real Data Analysis 22
6.1 Data Description . . . . . . . . . . . . . . . . . . . . . . . . . . . . 22
6.2 Analysis Results . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 23
7 Conclusion 26
Reference 27
Appendix 28
A Proof of Theorems 28
A.1 Proof of Theorem 1 . . . . . . . . . . . . . . . . . . . . . . . . . . . 30
A.2 Proof of Theorem 2 . . . . . . . . . . . . . . . . . . . . . . . . . . . 36
A.3 Proof of Theorem 3 . . . . . . . . . . . . . . . . . . . . . . . . . . . 39
A.4 Proof of Theorem 4 . . . . . . . . . . . . . . . . . . . . . . . . . . . 45
B Simulation and Real data results 53
B.1 Simulation results . . . . . . . . . . . . . . . . . . . . . . . . . . . . 53
B.2 Real data results . . . . . . . . . . . . . . . . . . . . . . . . . . . . 79
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