The design and implementation of N-of-1 trials_Chinese Journal of Evidence-Based Medicine

Authors：

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

1. Tianjin University of Traditional Chinese Medicine, Tianjin, 300193, P.R.China;
2. 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;
3. Key Laboratory of Chinese Internal Medicine of Ministry of Education and Beijing, Dongzhimen Hospital, Beijing University of Chinese Medicine, Beijing, 100700, P.R.China;

Corresponding author：

CHEN Jing, Email: cjshcsyc@126.com; SHANG Hongcai, Email: shanghongcai@foxmail.com

Keywords：

N-of-1 trial; Design; Implementation; Application

DOI：

10.7507/1672-2531.201701076

Video：

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N-of-1 trial design offers a methodologically sound approach to determining optimum treatment for an individual patient and solves some limitations of randomized controlled trials. This design could offer an efficient method of reaching a personal treatment regime tailored to suit individual needs and preferences. The paper introduces practical application, objects and the implementation process of N-of-1 trial, to explore its design points and implementation.

Citation： WANG Hui, ZHAI Jingbo, CHEN Jing, LI Jiang, SHANG Hongcai. The design and implementation of N-of-1 trials. Chinese Journal of Evidence-Based Medicine, 2017, 17(3): 364-368. doi: 10.7507/1672-2531.201701076 Copy

1.	Leber P. Not in our methods, but in our ignorance. Arch Gen Psychiatry, 2002, 59(3): 279-280.
2.	Grimley EJ. Evidence-based and evidence-based medicine. Age Ageing, 1995, 24(6): 461-463.
3.	Guyatt GH, Keller JL, Jaeschke R,et al. The n-of-1 randomized controlled trial: clinical usefulness. Our three-year experience. Ann Intern Med, 1990, 112(4): 293-299.
4.	Nikles CJ, Glasziou PP, Del Mar CB,et al. N-of-1 trials: practical tools for medication management. Aust Fam Physician, 2000, 29(11): 1108-1112.
5.	Nikles CJ, Clavarino AM, Del Mar CB. Using n-of-1 trials as a clinical tool to improve prescribing. Br J Gen Pract, 2005, 55(512): 175-180.
6.	傅鹰. 单病例随机对照治疗试验. 药物流行病学杂志, 1994, 4(5): 42-43.
7.	刘建平, 张玫, 杨闵, 等. 单病例随机对照试验设计在中医药研究中的应用. 中国中医药信息杂志, 2002, 9(6): 66-68.
8.	王辉, 陈静, 商洪才. 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.
9.	Lindberg G. Single case studies in clinical trials. Scand J Gastroenterol Suppl, 1988, 23(147): 30-32.
10.	Guyatt GH, Sackett D, Adachi J,et al. A clinician's guide for conducting randomized trials in individual patients. CMAJ, 1988, 139(6): 497-503.
11.	Faber A, Keizer RJ, van den Berg PB,et al. Use of double-blind placebo-controlled N-of-1 trials among stimulant-treated youths in the Netherlands: a descriptive study. Eur J Clin Pharmacol, 2007, 63(1): 57-63.
12.	Guyatt G, Sackett D, Taylor DW,et al. Determining optimal therapy-randomized trials in individual patients. N Engl J Med, 1986, 314(14): 889-892.
13.	McLeod RS, Taylor DW, Cohen Z,et al. Single-patient randomized clinical trial.Use in determining optimum treatment for patient with inflammation of Kock continent ileostomy reservoir. Lancet, 1986, 1(8483): 726-728.
14.	Mahon J, Laupacis A, Donner A,et al. Randomized study of n of 1 trials versus standard practice. BMJ, 1996, 312(7038): 1069-1074.
15.	March L, Irwig L, Schwarz J,et al. N of 1 trials comparing a non-steroidal anti-inflammatory drug with paracetamol in osteoarthritis. BMJ, 1994, 309(6961): 1041-1045.
16.	Woodfield R, Goodyear-Smith F, Arroll B. N-of-1 trials of quinine efficacy in skeletal muscle cramps of the leg. Br J Gen Pract, 2005, 55(512): 181-185.
17.	Chatellier G, Day M, Bobrie G,et al. Feasibility study of n-of-1 trials with blood pressure self-monitoring in hypertension. Hypertension, 1995, 25(2): 294-301.
18.	Jaeschke R, Adachi J, Guyatt G,et al. Clinical usefulness of amitriptyline in fibromyalgia:the results of 23 N-of-1 randomized controlled trials. J Rheumatol, 1991, 18(3): 447-451.
19.	Hoogwerf BJ. Amitriptyline treatment of painful diabetic neuropathy: an inadvertent single-patient clinical trial. Diabetes Care, 1985, 8(5): 526-527.
20.	Nuovo J, Ellsworth AJ, Larson EB. Treatment of atopic dermatitis with antihistamines: lessons from a single-patient randomized clinical trial. J Am Board Fam Pract, 1992, 5(2): 137-141.
21.	Dales RE, Lunau MA, Tierney MG. Chronic cough responsive to ibuprofen. Pharmacotherap, 1992, 12(4): 331-333.
22.	Estrada CA, Young MJ. Patient preferences for novel therapy: an N-of-1 trial of garlic in the treatment for hypertension. J Gen Intern Med, 1993, 8(11): 619.
23.	Guyatt GH, Heyting A, Jaeschke R,et al. N of 1 randomized trials for investigating new drugs. Contril Clin Trials, 1990, 11(2): 88-100.
24.	蹇文渊. 芪明颗粒治疗糖尿病视网膜病变的单病例随机对照试验研究. 成都: 成都中医药大学, 2013.
25.	田永明. 十味益元颗粒治疗化疗骨髓抑制的单病例随机对照试验. 北京: 中国中医科学院, 2010.
26.	Andrew LA, Stephen B, John MN. Use of an embedded N-of-1 trial to improve adherence and increase information from a clinical study. Contemp Clin Trials, 2005, 26(3): 397-401.
27.	刘建平. 单个病例随机对照试验的设计与应用. 中国中西医结合杂志, 2005, 25(3): 252-254.
28.	Guyatt G. N of l randomized trials: a commentary. J Clin Epidemiol, 2016, pii: S0895-4356(16): 30066.
29.	Price JD, Grimley EJ. N-of-1 randomized controlled trials: singularly useful in geriatric medicine. Age Ageing, 2002, 31(4): 227-232.
30.	Nikles CJ, Del Mar CB. Using n-of-1 trials as a clinical tool to improve prescribing. Br J Gen Pract, 2005, 55(512): 175-180.
31.	Madhok V, Fahey T. N-of-1 trials: an opportunity to tailor treatment in individual patients. Br J Gen Pract, 2005, 55(512): 172-174.
32.	Pope JE, Prashker M, Anderson J. The efficacy and cost effectiveness of N of 1 studies with diclofenac compared to standard treatment with nonsteroidal anti-inflammatory drugs in osteoarthritis. J Rheumatol, 2004, 31(1): 140-149.
33.	Wegman AC, van der Windt DA, Stalman WA,et al. Conducting research in individual patients: lessons learnt from two series of N-of-1 trials. BMC Family Practice, 2006, 7(1): 54.
34.	Guyatt G, Haynes B, Jaeschke R,et al. Introduction: the philosophy of evidence-based medicine. In users’ guides to the medical literature. Chicago: American Medical Association Press, 2002: 3-11.

1. Leber P. Not in our methods, but in our ignorance. Arch Gen Psychiatry, 2002, 59(3): 279-280.
2. Grimley EJ. Evidence-based and evidence-based medicine. Age Ageing, 1995, 24(6): 461-463.
3. Guyatt GH, Keller JL, Jaeschke R,et al. The n-of-1 randomized controlled trial: clinical usefulness. Our three-year experience. Ann Intern Med, 1990, 112(4): 293-299.
4. Nikles CJ, Glasziou PP, Del Mar CB,et al. N-of-1 trials: practical tools for medication management. Aust Fam Physician, 2000, 29(11): 1108-1112.
5. Nikles CJ, Clavarino AM, Del Mar CB. Using n-of-1 trials as a clinical tool to improve prescribing. Br J Gen Pract, 2005, 55(512): 175-180.
6. 傅鹰. 单病例随机对照治疗试验. 药物流行病学杂志, 1994, 4(5): 42-43.
7. 刘建平, 张玫, 杨闵, 等. 单病例随机对照试验设计在中医药研究中的应用. 中国中医药信息杂志, 2002, 9(6): 66-68.
8. 王辉, 陈静, 商洪才. 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.
9. Lindberg G. Single case studies in clinical trials. Scand J Gastroenterol Suppl, 1988, 23(147): 30-32.
10. Guyatt GH, Sackett D, Adachi J,et al. A clinician's guide for conducting randomized trials in individual patients. CMAJ, 1988, 139(6): 497-503.
11. Faber A, Keizer RJ, van den Berg PB,et al. Use of double-blind placebo-controlled N-of-1 trials among stimulant-treated youths in the Netherlands: a descriptive study. Eur J Clin Pharmacol, 2007, 63(1): 57-63.
12. Guyatt G, Sackett D, Taylor DW,et al. Determining optimal therapy-randomized trials in individual patients. N Engl J Med, 1986, 314(14): 889-892.
13. McLeod RS, Taylor DW, Cohen Z,et al. Single-patient randomized clinical trial.Use in determining optimum treatment for patient with inflammation of Kock continent ileostomy reservoir. Lancet, 1986, 1(8483): 726-728.
14. Mahon J, Laupacis A, Donner A,et al. Randomized study of n of 1 trials versus standard practice. BMJ, 1996, 312(7038): 1069-1074.
15. March L, Irwig L, Schwarz J,et al. N of 1 trials comparing a non-steroidal anti-inflammatory drug with paracetamol in osteoarthritis. BMJ, 1994, 309(6961): 1041-1045.
16. Woodfield R, Goodyear-Smith F, Arroll B. N-of-1 trials of quinine efficacy in skeletal muscle cramps of the leg. Br J Gen Pract, 2005, 55(512): 181-185.
17. Chatellier G, Day M, Bobrie G,et al. Feasibility study of n-of-1 trials with blood pressure self-monitoring in hypertension. Hypertension, 1995, 25(2): 294-301.
18. Jaeschke R, Adachi J, Guyatt G,et al. Clinical usefulness of amitriptyline in fibromyalgia:the results of 23 N-of-1 randomized controlled trials. J Rheumatol, 1991, 18(3): 447-451.
19. Hoogwerf BJ. Amitriptyline treatment of painful diabetic neuropathy: an inadvertent single-patient clinical trial. Diabetes Care, 1985, 8(5): 526-527.
20. Nuovo J, Ellsworth AJ, Larson EB. Treatment of atopic dermatitis with antihistamines: lessons from a single-patient randomized clinical trial. J Am Board Fam Pract, 1992, 5(2): 137-141.
21. Dales RE, Lunau MA, Tierney MG. Chronic cough responsive to ibuprofen. Pharmacotherap, 1992, 12(4): 331-333.
22. Estrada CA, Young MJ. Patient preferences for novel therapy: an N-of-1 trial of garlic in the treatment for hypertension. J Gen Intern Med, 1993, 8(11): 619.
23. Guyatt GH, Heyting A, Jaeschke R,et al. N of 1 randomized trials for investigating new drugs. Contril Clin Trials, 1990, 11(2): 88-100.
24. 蹇文渊. 芪明颗粒治疗糖尿病视网膜病变的单病例随机对照试验研究. 成都: 成都中医药大学, 2013.
25. 田永明. 十味益元颗粒治疗化疗骨髓抑制的单病例随机对照试验. 北京: 中国中医科学院, 2010.
26. Andrew LA, Stephen B, John MN. Use of an embedded N-of-1 trial to improve adherence and increase information from a clinical study. Contemp Clin Trials, 2005, 26(3): 397-401.
27. 刘建平. 单个病例随机对照试验的设计与应用. 中国中西医结合杂志, 2005, 25(3): 252-254.
28. Guyatt G. N of l randomized trials: a commentary. J Clin Epidemiol, 2016, pii: S0895-4356(16): 30066.
29. Price JD, Grimley EJ. N-of-1 randomized controlled trials: singularly useful in geriatric medicine. Age Ageing, 2002, 31(4): 227-232.
30. Nikles CJ, Del Mar CB. Using n-of-1 trials as a clinical tool to improve prescribing. Br J Gen Pract, 2005, 55(512): 175-180.
31. Madhok V, Fahey T. N-of-1 trials: an opportunity to tailor treatment in individual patients. Br J Gen Pract, 2005, 55(512): 172-174.
32. Pope JE, Prashker M, Anderson J. The efficacy and cost effectiveness of N of 1 studies with diclofenac compared to standard treatment with nonsteroidal anti-inflammatory drugs in osteoarthritis. J Rheumatol, 2004, 31(1): 140-149.
33. Wegman AC, van der Windt DA, Stalman WA,et al. Conducting research in individual patients: lessons learnt from two series of N-of-1 trials. BMC Family Practice, 2006, 7(1): 54.
34. Guyatt G, Haynes B, Jaeschke R,et al. Introduction: the philosophy of evidence-based medicine. In users’ guides to the medical literature. Chicago: American Medical Association Press, 2002: 3-11.

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The design and implementation of N-of-1 trials

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