Abstract
N-of-1 trials and review articles have recently been published in the areas of chronic pain, pediatrics, palliative care, complementary and alternative medicine, rare diseases, patient-centered care, the behavioral sciences and genomics. These are briefly reviewed and the current place of N-of-1 trials discussed. The chapter concludes with a vision for the future of N-of-1 trials.
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References
Berde CB, Walco GA, Krane EJ, Anand KJ, Aranda JV, Craig KD, Dampier CD, Finkel JC, Grabois M, Johnston C, Lantos J, Lebel A, Maxwell LG, McGrath P, Oberlander TF, Schanberg LE, Stevens B, Taddio A, Von Baeyer CL, Yaster M, Zempsky WT (2012) Pediatric analgesic clinical trial designs, measures, and extrapolation: report of an FDA scientific workshop. Pediatrics 129(2):354–364. doi:10.1542/peds.2010-3591, Epub 2012 Jan 16
Bush A. Montelukast in paediatric asthma: where we are now and what still needs to be done? 7 2014 Dec 11. pii: S1526-0542(14)00131-6. doi: 10.1016/j.prrv.2014.10.007
Camfield P, Gordon K, Dooley J, Camfield C (1996) Melatonin appears ineffective in children with intellectual deficits and fragmented sleep: six "N of 1" trials. J Child Neurol 11(4):341–343
Cook AM, Finlay IG, Butler-Keating RJ (2002) Recruiting into palliative care trials: lessons learnt from a feasibility study. Palliat Med 16:163–165
Coxeter P, Schluter P, Eastwood H, Nikles J, Glasziou P (2003) Valerian does not reduce symptoms for patients with chronic insomnia in general practice. Complement Ther Med 11(4):215–222
Currow DC, Plummer JL, Kutner JS, Samsa GP, Abernethy AP (2012) Analyzing phase III studies in hospice/palliative care. A solution that sits between intention-to-treat and per protocol analyses: the palliative-modified ITT analysis. J Pain Symptom Manag 44(4):595–603
Davidson KW, Peacock J, Kronish IM, Edmondson D (2014) Personalizing behavioral interventions through single-patient (N-of-1) trials. Soc Personal Psychol Compass 8(8):408–421
Davis MP, Mitchell GK (2012) Topics in research: structuring studies in palliative care. Curr Opin Support Palliat Care 6(4):483–489. doi:10.1097/SPC.0b013e32835843d7
Duan N, Kravitz RL, Schmid CH (2013) Single-patient (N-of-1) trials: a pragmatic clinical decision methodology for patient-centered comparative effectiveness research. J Clin Epidemiol 66(8 Suppl):S21–S28. doi:10.1016/j.jclinepi.2013.04.006
Duggan CM, Mitchell G, Nikles CJ, Glasziou PP, Del Mar CB, Clavarino A (2000) Managing ADHD in general practice. N of 1 trials can help! Aust Fam Physician 29(12):1205–1209
Evans JJ, Gast DL, Perdices M, Manolov R (2014) Single case experimental designs: introduction to a special issue of neuropsychological rehabilitation. Neuropsychol Rehabil 24(3–4):305–314. doi:10.1080/09602011.2014.903198, Epub 2014 Apr 25
Faber A, Keizer RJ, van den Berg PB, de Jong-van den Berg LT, Tobi H (2007) Use of double-blind placebo-controlled N-of-1 trials among stimulant-treated youths in the Netherlands: a descriptive study. Eur J Clin Pharmacol 63(1):57–63, Epub 2006 Nov 18
Facey K, Granados A, Guyatt G, Kent A, Shah N, van der Wilt GJ, Wong-Rieger D (2014) Generating health technology assessment evidence for rare diseases. Int J Technol Assess Health Care 19:1–7
Gewandter JS, Dworkin RH, Turk DC, McDermott MP, Baron R, Gastonguay MR, Gilron I, Katz NP, Mehta C, Raja SN, Senn S, Taylor C, Cowan P, Desjardins P, Dimitrova R, Dionne R, Farrar JT, Hewitt DJ, Iyengar S, Jay GW, Kalso E, Kerns RD, Leff R, Leong M, Petersen KL, Ravina BM, Rauschkolb C, Rice AS, Rowbotham MC, Sampaio C, Sindrup SH, Stauffer JW, Steigerwald I, Stewart J, Tobias J, Treede RD, Wallace M, White RE (2014) Research designs for proof-of-concept chronic pain clinical trials: IMMPACT recommendations. Pain 155(9):1683–1695. doi:10.1016/j.pain.2014.05.025, Epub 2014 May 24
Greenhill LL, Jensen PS, Abikoff H, Blumer JL, Deveaugh-Geiss J, Fisher C, Hoagwood K, Kratochvil CJ, Lahey BB, Laughren T, Leckman J, Petti TA, Pope K, Shaffer D, Vitiello B, Zeanah C (2003) Developing strategies for psychopharmacological studies in preschool children. J Am Acad Child Adolesc Psychiatry 42(4):406–414, Review
Gupta S, Faughnan ME, Tomlinson GA, Bayoumi AM (2011) A framework for applying unfamiliar trial designs in studies of rare diseases. J Clin Epidemiol 64(10):1085–1094. doi:10.1016/j.jclinepi.2010.12.019, Epub 2011 May 6
Hermet R, Burucia B, Sentilles-Monkam A (2002) The need for evidence-based proof in palliative care. Eur J Pall Care 9:104–107
Huang H, Yang P, Xue J, Tang J, Ding L, Ma Y, Wang J, Guyatt GH, Vanniyasingam T, Zhang Y (2014) Evaluating the individualized treatment of traditional Chinese medicine: a pilot study of N-of-1 trials. Evid Based Complement Alternat Med 2014:148730. doi:10.1155/2014/148730, Epub 2014 Nov 11
Huber AM, Tomlinson GA, Koren G, Feldman BM (2007) Amitriptyline to relieve pain in juvenile idiopathic arthritis: a pilot study using Bayesian metaanalysis of multiple N-of-1 clinical trials. J Rheumatol 34(5):1125–1132, Epub 2007 Apr 15
Kaput J, Morine M (2012) Discovery-based nutritional systems biology: developing N-of-1 nutrigenomic research. Int J Vitam Nutr Res 82(5):333–341. doi:10.1024/0300-9831/a000128
Kent MA, Camfield CS, Camfield PR (1999) Double-blind methylphenidate trials: practical, useful, and highly endorsed by families. Arch Pediatr Adolesc Med 153(12):1292–1296
Lee S, Lim N, Choi SM, Kim S (2012) Validation study of Kim’s sham needle by measuring facial temperature: an N-of-1 randomized double-blind placebo-controlled clinical trial. Evid Based Complement Alternat Med 2012:507937. doi:10.1155/2012/507937, Epub 2012 Mar 6
Li J, Tian J, Ma B, Yang K (2013) N-of-1 trials in china. Complement Ther Med 21(3):190–194. doi:10.1016/j.ctim.2013.01.003, Epub 2013 Feb 18
Lillie EO, Patay B, Diamant J, Issell B, Topol EJ, Schork NJ (2011) The N-of-1 clinical trial: the ultimate strategy for individualizing medicine? Per Med 8(2):161–173
Nathan PC, Tomlinson G, Dupuis LL, Greenberg ML, Ota S, Bartels U, Feldman BM (2006) A pilot study of ondansetron plus metopimazine vs. Ondansetron Monotherapy in children receiving highly emetogenic chemotherapy: a Bayesian randomized serial N-of-1 trials design. Support Care Cancer 14(3):268–276, Epub 2005 Jul 29
Nikles CJ, Clavarino AM, Del Mar CB (2005) Using N-of-1 trials as a clinical tool to improve prescribing. Br J Gen Pract 55(512):175–180
Nikles CJ, Mitchell GK, Del Mar CB, Clavarino AM, McNairn N (2006) An n-of-1 trial service in clinical practice: testing the effectiveness of stimulants for attention-deficit/hyperactivity disorder. Pediatrics 117(6):2040–2046
Nikles CJ, McKinlay L, Mitchell GK, Carmont SA, Senior HE, Waugh MC, Epps A, Schluter PJ, Lloyd OT (2014) Aggregated n-of-1 trials of central nervous system stimulants versus placebo for paediatric traumatic brain injury–a pilot study. Trials 15:54. doi:10.1186/1745-6215-15-54
Sung L, Feldman BM (2006) N-of-1 trials: innovative methods to evaluate complementary and alternative medicines in pediatric cancer. J Pediatr Hematol Oncol 28(4):263–266
Sung L, Tomlinson GA, Greenberg ML, Koren G, Judd P, Ota S, Feldman BM (2007) Serial controlled N-of-1 trials of topical vitamin E as prophylaxis for chemotherapy-induced oral mucositis in paediatric patients. Eur J Cancer 43(8):1269–1275, Epub 2007 Mar 23
Suri R (2005) The use of human deoxyribonuclease (rhDNase) in the management of cystic fibrosis. BioDrugs 19(3):135–144
Suri R, Metcalfe C, Wallis C, Bush A (2004) Predicting response to rhDNase and hypertonic saline in children with cystic fibrosis. Pediatr Pulmonol 37(4):305–310
Taragin D, Berman S, Zelnik N, Karni A, Tirosh E (2013) Parents’ attitudes toward methylphenidate using n-of-1 trial: a pilot study. Atten Defic Hyperact Disord 5(2):105–109. doi:10.1007/s12402-012-0099-x. Epub 2012 Dec 16
Tate RL, McDonald S, Perdices M, Togher L, Schultz R, Savage S (2008) Rating the methodological quality of single-subject designs and n-of-1 trials: introducing the single-case experimental design (SCED) scale. Neuropsychol Rehabil 18(4):385–401. doi:10.1080/09602010802009201
Tate RL, Perdices M, Rosenkoetter U, Wakim D, Godbee K, Togher L, McDonald S (2013) Revision of a method quality rating scale for single-case experimental designs and n-of-1 trials: the 15-item risk of bias in N-of-1 trials (RoBiNT) scale. Neuropsychol Rehabil 23(5):619–638. doi:10.1080/09602011.2013.824383, Epub 2013 Sep 9
Tate RL, Perdices M, McDonald S, Togher L, Rosenkoetter U (2014) The design, conduct and report of single-case research: resources to improve the quality of the neurorehabilitation literature. Neuropsychol Rehabil 24(3–4):315–331. doi:10.1080/09602011.2013.875043, Epub 2014 Apr 7
Yuhong H, Qian L, Yu L, Yingqiang Z, Yanfen L, Shujing Y, Shufang Q, Lanjun S, Shuxuan Z, Baohe W (2013) An n-of-1 trial service in clinical practice: testing the effectiveness of Liuwei Dihuang decoction for kidney-Yin defisciency syndrome. Evid Based Complement Alternat Med 2013:827915. doi:10.1155/2013/827915, Epub 2013 Sep 23
Zucker DR, Schmid CH, McIntosh MW, D’Agostino RB, Selker HP, Lau J (1997) Combining single patient (N-of-1) trials to estimate population treatment effects and to evaluate individual patient responses to treatment. J Clin Epidemiol 50(4):401–410
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Nikles, J. (2015). Where Are N-of-1 Trials Headed?. In: Nikles, J., Mitchell, G. (eds) The Essential Guide to N-of-1 Trials in Health. Springer, Dordrecht. https://doi.org/10.1007/978-94-017-7200-6_17
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DOI: https://doi.org/10.1007/978-94-017-7200-6_17
Publisher Name: Springer, Dordrecht
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