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.	陳新林, 李海文, 劉鳳斌, 等. 單病例隨機對照試驗的設計和評價方法. 中國循證醫學雜志, 2016, 16(2): 242-245.
4.	陳新林, 黃海茵, 張長榮, 等. 2015版CENT聲明簡介: 單病例隨機對照試驗報告指南. 中國循證醫學雜志, 2016, 16(7): 860-863.
5.	陳新林, 陳平雁. N-of-1無殘留效應定量數據混合效應模型的模擬研究. 中國衛生統計, 2013, 30(3): 347-350.
6.	田金徽, 楊克虎. 單病例隨機對照試驗的研究現狀與進展. 蘭州大學學報(醫學版), 2016, 42(3): 52-56.
7.	陳新林, 趙菲, 胡月, 等. 探索適合中醫藥N-of-1試驗的設計方案及模型. 廣州中醫藥大學學報, 2018, 35(1): 172-175.
8.	王梅, 王建華, 張抗, 等. 中醫藥療效評價隊列研究的方法學質量評價. 中醫雜志, 2016, 57(16): 1379-1383.
9.	翟靜波, 李江, 商洪才, 等. 貝葉斯單病例隨機對照試驗簡介. 中國循證醫學雜志, 2017, 17(7): 848-851.
10.	李江, 翟靜波, 商洪才, 等. 單病例隨機對照試驗的起源與發展. 中國循證醫學雜志, 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. 陳新林, 李海文, 劉鳳斌, 等. 單病例隨機對照試驗的設計和評價方法. 中國循證醫學雜志, 2016, 16(2): 242-245.
4. 陳新林, 黃海茵, 張長榮, 等. 2015版CENT聲明簡介: 單病例隨機對照試驗報告指南. 中國循證醫學雜志, 2016, 16(7): 860-863.
5. 陳新林, 陳平雁. N-of-1無殘留效應定量數據混合效應模型的模擬研究. 中國衛生統計, 2013, 30(3): 347-350.
6. 田金徽, 楊克虎. 單病例隨機對照試驗的研究現狀與進展. 蘭州大學學報(醫學版), 2016, 42(3): 52-56.
7. 陳新林, 趙菲, 胡月, 等. 探索適合中醫藥N-of-1試驗的設計方案及模型. 廣州中醫藥大學學報, 2018, 35(1): 172-175.
8. 王梅, 王建華, 張抗, 等. 中醫藥療效評價隊列研究的方法學質量評價. 中醫雜志, 2016, 57(16): 1379-1383.
9. 翟靜波, 李江, 商洪才, 等. 貝葉斯單病例隨機對照試驗簡介. 中國循證醫學雜志, 2017, 17(7): 848-851.
10. 李江, 翟靜波, 商洪才, 等. 單病例隨機對照試驗的起源與發展. 中國循證醫學雜志, 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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