Sample size estimation for single case randomized controlled trial (N-of-1 trial)_Chinese Journal of Evidence-Based Medicine

Authors：

 CHEN Xinlin

Basic Medical College, Guangzhou University of Chinese Medicine, Guangzhou, 510006, P.R.China;

Corresponding author：

CHEN Xinlin, Email: chenxlsums@126.com

Keywords：

Single case randomized controlled trial; N-of-1 trial; Sample size; Fixed model; Random model

DOI：

10.7507/1672-2531.201901075

Video：

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With increasing amount of attention being paid to single case randomized controlled trial (N-of-1 trials), sample size estimation has become an important issue for clinical researchers. This paper mainly introduces the model and hypothesis of N-of-1 trials. Based on the hypothetical model, sample size estimation methods of fixed model and random model are proposed. The premises of the model application, formulas and examples are then given. It is expected in case of conduction N-of-1 trials, the correct methods are used to estimate sample size and improve the research quality of N-of-1 trials.

Citation： CHEN Xinlin. Sample size estimation for single case randomized controlled trial (N-of-1 trial). Chinese Journal of Evidence-Based Medicine, 2019, 19(7): 871-874. doi: 10.7507/1672-2531.201901075 Copy

1.	Shamseer L, Sampson M, Bukutu C, et al. CONSORT extension for reporting N-of-1 trials (CENT) 2015: Explanation and elaboration. BMJ, 2015, 350: h1793.
2.	Li J, Gao W, Punja S, et al. Reporting quality of N-of-1 trials published between 1985 and 2013: a systematic review. J Clin Epidemiol, 2016, 76: 57-64.
3.	陈新林, 李海文, 刘凤斌, 等. 单病例随机对照试验的设计和评价方法. 中国循证医学杂志, 2016, 16(2): 242-245.
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5.	陈新林, 陈平雁. N-of-1无残留效应定量数据混合效应模型的模拟研究. 中国卫生统计, 2013, 30(3): 347-350.
6.	田金徽, 杨克虎. 单病例随机对照试验的研究现状与进展. 兰州大学学报(医学版), 2016, 42(3): 52-56.
7.	陈新林, 赵菲, 胡月, 等. 探索适合中医药N-of-1试验的设计方案及模型. 广州中医药大学学报, 2018, 35(1): 172-175.
8.	王梅, 王建华, 张抗, 等. 中医药疗效评价队列研究的方法学质量评价. 中医杂志, 2016, 57(16): 1379-1383.
9.	翟静波, 李江, 商洪才, 等. 贝叶斯单病例随机对照试验简介. 中国循证医学杂志, 2017, 17(7): 848-851.
10.	李江, 翟静波, 商洪才, 等. 单病例随机对照试验的起源与发展. 中国循证医学杂志, 2017, 17(2): 235-238.
11.	Senn S. Cross-over trials in clinical research (2_nd Edition). New York: John Wiley & Sons Ltd, 2002.
12.	Chow S, Shao J, Wang H. Sample size calculations in clinical research (2nd Edition). London: Chapman and Hall/CRC, 2008.
13.	Senn S. Sample size considerations for N-of-1 trials. Stat Methods Med Res, 2019, 28(2): 372-383.
14.	Xie T, Yu Z. N-of-1 Design and its applications to personalized treatment studies. Stat Biosci, 2017, 9(2): 662-675.
15.	Clough AJ, Hilmer SN, Naismith SL, et al. N-of-1 trials for assessing the effects of deprescribing medications on short-term clinical outcomes in older adults: a systematic review. J Clin Epidemiol, 2018, 93: 112-119.
16.	Araujo A, Julious S, Senn S. Understanding variation in sets of N-of-1 trials. PLoS One, 2016, 11(12): e0167167.
17.	Chen X, Chen P. A comparison of four methods for the analysis of N-of-1 trials. PLoS One, 2014, 9(2): e87752.
18.	Zucker DR, Ruthazer R, Schmid CH. Individual (N-of-1) trials can be combined to give population comparative treatment effect estimates: methodologic considerations. J Clin Epidemiol, 2010, 63(12): 1312-1323.
19.	Zucker DR, Schmid CH, McIntosh MW, et al. Combining single patient (N-of-1) trials to estimate population treatment effects and to evaluate individual patient responses to treatment. J Clin Epidemiol, 1997, 50(4): 401-410.
20.	O'Brien RG, Muller KE. Applied analysis of variance in behavioral science. New York: Marcel Decker, 1993: 297-344.

1. Shamseer L, Sampson M, Bukutu C, et al. CONSORT extension for reporting N-of-1 trials (CENT) 2015: Explanation and elaboration. BMJ, 2015, 350: h1793.
2. Li J, Gao W, Punja S, et al. Reporting quality of N-of-1 trials published between 1985 and 2013: a systematic review. J Clin Epidemiol, 2016, 76: 57-64.
3. 陈新林, 李海文, 刘凤斌, 等. 单病例随机对照试验的设计和评价方法. 中国循证医学杂志, 2016, 16(2): 242-245.
4. 陈新林, 黄海茵, 张长荣, 等. 2015版CENT声明简介: 单病例随机对照试验报告指南. 中国循证医学杂志, 2016, 16(7): 860-863.
5. 陈新林, 陈平雁. N-of-1无残留效应定量数据混合效应模型的模拟研究. 中国卫生统计, 2013, 30(3): 347-350.
6. 田金徽, 杨克虎. 单病例随机对照试验的研究现状与进展. 兰州大学学报(医学版), 2016, 42(3): 52-56.
7. 陈新林, 赵菲, 胡月, 等. 探索适合中医药N-of-1试验的设计方案及模型. 广州中医药大学学报, 2018, 35(1): 172-175.
8. 王梅, 王建华, 张抗, 等. 中医药疗效评价队列研究的方法学质量评价. 中医杂志, 2016, 57(16): 1379-1383.
9. 翟静波, 李江, 商洪才, 等. 贝叶斯单病例随机对照试验简介. 中国循证医学杂志, 2017, 17(7): 848-851.
10. 李江, 翟静波, 商洪才, 等. 单病例随机对照试验的起源与发展. 中国循证医学杂志, 2017, 17(2): 235-238.
11. Senn S. Cross-over trials in clinical research (2_nd Edition). New York: John Wiley & Sons Ltd, 2002.
12. Chow S, Shao J, Wang H. Sample size calculations in clinical research (2nd Edition). London: Chapman and Hall/CRC, 2008.
13. Senn S. Sample size considerations for N-of-1 trials. Stat Methods Med Res, 2019, 28(2): 372-383.
14. Xie T, Yu Z. N-of-1 Design and its applications to personalized treatment studies. Stat Biosci, 2017, 9(2): 662-675.
15. Clough AJ, Hilmer SN, Naismith SL, et al. N-of-1 trials for assessing the effects of deprescribing medications on short-term clinical outcomes in older adults: a systematic review. J Clin Epidemiol, 2018, 93: 112-119.
16. Araujo A, Julious S, Senn S. Understanding variation in sets of N-of-1 trials. PLoS One, 2016, 11(12): e0167167.
17. Chen X, Chen P. A comparison of four methods for the analysis of N-of-1 trials. PLoS One, 2014, 9(2): e87752.
18. Zucker DR, Ruthazer R, Schmid CH. Individual (N-of-1) trials can be combined to give population comparative treatment effect estimates: methodologic considerations. J Clin Epidemiol, 2010, 63(12): 1312-1323.
19. Zucker DR, Schmid CH, McIntosh MW, et al. Combining single patient (N-of-1) trials to estimate population treatment effects and to evaluate individual patient responses to treatment. J Clin Epidemiol, 1997, 50(4): 401-410.
20. O'Brien RG, Muller KE. Applied analysis of variance in behavioral science. New York: Marcel Decker, 1993: 297-344.

Chinese Journal of Evidence-Based Medicine

Sample size estimation for single case randomized controlled trial (N-of-1 trial)

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