利用半母數模型分析三臂非劣性試驗

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姓名

余岱錡(Tai-Chi Yu) 查詢紙本館藏

畢業系所

統計研究所

論文名稱

利用半母數模型分析三臂非劣性試驗

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至系統瀏覽論文 (2026-7-1以後開放)

摘要(中)

本篇的研究目的為在三臂非劣性試驗下，計算三組治療組不同比例的樣本數，探討在何種比例下有最佳樣本數，以及最佳樣本數的檢定力是否有達到我們所設定的目標。三臂非劣性試驗包含實驗組、對照組和安慰劑組，也稱為黃金標準試驗，用於右設限資料，其中考慮到行政設限和失去追蹤設限，將資料應用在不同的半母數模型進行模擬，本研究採用的半母數模型包括Cox迴歸模型、AFT模型和PO模型(Proportional Odds Model)在相同條件下做比較。我們以三組治療組來當作共變異數，使用模型來估計迴歸係數，得以了解治療組之間的藥效差異，再利用大樣本的分佈計算樣本數與檢定力。最後，我們將發展的方法運用在膀胱癌復發的研究上。

摘要(英)

The purpose of this study is to calculate the sample sizes for different allocation to three treatment groups in a three-arm non-inferiority trial. The aim is to determine the optimal sample size for each allocation and assess whether the achieved power meets our predefined target. The three-arm non-inferiority trial consists of an experimental group, a control group, and a placebo group, also known as a gold standard trial. It is designed for right-censored survival data, considering administrative censoring and lost to follow-up. The semiparametric models employed in this study include the Cox proportional hazards model, the accelerated failure time model, and the proportional odds model, which are compared under the same conditions. The three treatment groups are treated as covariates, and the models are used to estimate the coefficients, providing insights into the differences in treatment effects among the groups. The sample sizes and desired power are calculated based on the distribution of a large sample. Finally, the developed methodology is applied to a study on bladder cancer recurrence.

關鍵字(中)

★ 三臂非劣性試驗
★ Proportional Odds模型
★ 半參數
★ 最佳樣本數

關鍵字(英)

★ optimal sample size
★ proportional odds model
★ semiparametric
★ three-arm non-inferiority trial

論文目次

第1章緒論 1
第2章研究方法 14
第3章模擬研究 23
第4章資料分析 40
第5章結論與討論 48

參考文獻

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指導教授

曾議寬(Yi-Kuan Tseng)

審核日期

2023-7-11

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