Statistical approaches for analyzing data from N-of-1 trials_Chinese Journal of Evidence-Based Medicine

Authors：

ZHAI Jingbo ¹ , SHANG Hongcai ² , LI Jiang ³ , WANG Hui ¹ ,  DAI Min ³ ,  CHEN Jing ¹

1. Tianjin University of Traditional Chinese Medicine, Tianjin, 300193, P.R.China;
2. Key Laboratory of Chinese Internal Medicine of Ministry of Education and Beijing, Dongzhimen Hospital, Beijing University of Chinese Medicine, Beijing, 100700, P.R.China;
3. National Cancer Center/Program Office for Cancer Screening in Urban, Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, 100021, P.R.China;

Corresponding author：

DAI Min, Email: daimin2002@hotmail.com; CHEN Jing, Email: cjshcsyc@126.com

Keywords：

N-of-1 trial; Statistical analysis

DOI：

10.7507/1672-2531.201701077

Video：

Export PDF Favorites Scan Get Citation

Abstract Full text Figures/Tables Video References Cited by

An N-of-1 trial was conducted in a single patient. Statistical analysis is one of the most important parts of N-of-1 trials. The methods of statistical analysis for N-of-1 trials were reported in some reviews. However, there was still a lack of comparative analysis of these methods. In this study, we introduced the characteristics of statistical methods commonly used as well as some statistical problems which should be paid attention in N-of-1 trials. It is useful to provide some reference for statistical methods in order to high quality N-of-1 trials.

Citation： ZHAI Jingbo, SHANG Hongcai, LI Jiang, WANG Hui, DAI Min, CHEN Jing. Statistical approaches for analyzing data from N-of-1 trials. Chinese Journal of Evidence-Based Medicine, 2017, 17(4): 494-496. doi: 10.7507/1672-2531.201701077 Copy

1.	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.
2.	Gabler NB, Duan N, Vohra S, et al. N-of-1 trials in the medical literature: a systematic review. Med Care, 2011, 49(8): 761-768.
3.	刘志刚, 柴程芝, 朱欣佚, 等. 葛根汤治疗原发性痛经的多基线单病例随机对照试验. 中华中医药杂志, 2015, 30(7): 2473-2476.
4.	张大铮. 基于单病例随机对照试验设计的中医药个体疗效评价模式与策略研究. 成都: 成都中医药大学, 2013.
5.	陈炜炜, 李宁. 针刺治疗脊髓损伤后下肢痉挛: 单病例随机对照试验. 中国针灸, 2010, 30(6): 473-475.
6.	Huang YH, Liu Q, Liu Y, et al. An n-of-1 trial service in clinical practice: testing the effectiveness of liuwei dihuang decoction for kidney-yin deficiency syndrome. Evid Based Complement Alternat Med, 2013, 2013: 827915.
7.	王辉. 单病例随机对照试验设计在中医药临床研究的探索与实践. 中华中医药杂志, 2010, 25(11): 1823-1828.
8.	Punja S, Schmid CH, Hartling L, et al. To meta-analyze or not to meta-analyze? A combined meta-analysis of N-of-1 trial data with RCT data on amphetamines and methylphenidate for pediatric ADHD. J Clin Epidemiol, 2016, 76: 76-81.
9.	Jingbo Z, Hongbo C, Ming R, et al. Reporting of core items in hierarchical Bayesian analysis for aggregating N-of-1 trials to estimate population treatment effects is suboptimal. J Clin Epidemiol, 2016, 76: 99-107.
10.	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.
11.	Senior HE, Mitchell GK, Nikles J, et al. Using aggregated single patient (N-of-1) trials to determine the effectiveness of psychostimulants to reduce fatigue in advanced cancer patients: a rationale and protocol. BMC Palliat Care, 2013, 12(1): 17.
12.	Stunnenberg BC, Woertman W, Raaphorst J, et al. Combined N-of-1 trials to investigate mexiletine in non-dystrophic myotonia using a Bayesian approach: study rationale and protocol. BMC Neurol, 2015, 15(1): 43.
13.	Gordon G, David S, Jonathan A, et al. A clinician’s guide for conducting randomized trials in individual patients. CMAJ, 1988, 139(6): 497-503.

1. 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.
2. Gabler NB, Duan N, Vohra S, et al. N-of-1 trials in the medical literature: a systematic review. Med Care, 2011, 49(8): 761-768.
3. 刘志刚, 柴程芝, 朱欣佚, 等. 葛根汤治疗原发性痛经的多基线单病例随机对照试验. 中华中医药杂志, 2015, 30(7): 2473-2476.
4. 张大铮. 基于单病例随机对照试验设计的中医药个体疗效评价模式与策略研究. 成都: 成都中医药大学, 2013.
5. 陈炜炜, 李宁. 针刺治疗脊髓损伤后下肢痉挛: 单病例随机对照试验. 中国针灸, 2010, 30(6): 473-475.
6. Huang YH, Liu Q, Liu Y, et al. An n-of-1 trial service in clinical practice: testing the effectiveness of liuwei dihuang decoction for kidney-yin deficiency syndrome. Evid Based Complement Alternat Med, 2013, 2013: 827915.
7. 王辉. 单病例随机对照试验设计在中医药临床研究的探索与实践. 中华中医药杂志, 2010, 25(11): 1823-1828.
8. Punja S, Schmid CH, Hartling L, et al. To meta-analyze or not to meta-analyze? A combined meta-analysis of N-of-1 trial data with RCT data on amphetamines and methylphenidate for pediatric ADHD. J Clin Epidemiol, 2016, 76: 76-81.
9. Jingbo Z, Hongbo C, Ming R, et al. Reporting of core items in hierarchical Bayesian analysis for aggregating N-of-1 trials to estimate population treatment effects is suboptimal. J Clin Epidemiol, 2016, 76: 99-107.
10. 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.
11. Senior HE, Mitchell GK, Nikles J, et al. Using aggregated single patient (N-of-1) trials to determine the effectiveness of psychostimulants to reduce fatigue in advanced cancer patients: a rationale and protocol. BMC Palliat Care, 2013, 12(1): 17.
12. Stunnenberg BC, Woertman W, Raaphorst J, et al. Combined N-of-1 trials to investigate mexiletine in non-dystrophic myotonia using a Bayesian approach: study rationale and protocol. BMC Neurol, 2015, 15(1): 43.
13. Gordon G, David S, Jonathan A, et al. A clinician’s guide for conducting randomized trials in individual patients. CMAJ, 1988, 139(6): 497-503.

Previous Article
A brief introduction of scoping review

Chinese Journal of Evidence-Based Medicine

Statistical approaches for analyzing data from N-of-1 trials

Abstract Full text Figures/Tables Video References Cited by

Previous Article

Format

Content