Standard 4: Determining adequate sample sizes

Pediatrics

Volumn 129, Issue SUPPL. 3, 2012, Pages

  Van Der Tweel, Ingeborg ^a   Askie, Lisa ^b   Vandermeer, Ben ^c   Ellenberg, Susan ^d   Fernandes, Ricardo M ^e   Saloojee, Haroon ^f   Bassler, Dirk ^g   Altman, Douglas G ^h   Offringa, Martin ⁱ   Van Der Lee, Johanna H ^j  

^a UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^b UNIVERSITY OF SYDNEY   (Australia)

^c UNIVERSITY OF ALBERTA   (Canada)

^d UNIVERSITY OF PENNSYLVANIA   (United States)

^e HOSPITAL DE SANTA MARIA   (Portugal)

^f UNIVERSITY OF THE WITWATERSRAND   (South Africa)

^g UNIVERSITY HOSPITAL TÜBINGEN   (Germany)

^h UNIVERSITY OF OXFORD   (United Kingdom)

ⁱ HOSPITAL FOR SICK CHILDREN RESEARCH INSTITUTE   (Canada)

^j EMMA CHILDREN S HOSPITAL   (Netherlands)

more..

Author keywords

Bayesian methods; Nuisance estimates; Pediatric; Sample size; StaR child health

Indexed keywords

CORTICOSTEROID; DEXAMETHASONE; HERBACEOUS AGENT; METHYLXANTHINE DERIVATIVE; PLACEBO;

ANALYTICAL ERROR; APNEA; ARTIFICIAL VENTILATION; BAYES THEOREM; CALCULATION; CHEMOTHERAPY INDUCED NAUSEA AND VOMITING; CHILD HEALTH CARE; CHILDHOOD OBESITY; CLINICAL RESEARCH; CLINICAL TRIAL (TOPIC); CONFERENCE PAPER; DRUG EFFICACY; EFFECT SIZE; GINGER; HUMAN; LOWER RESPIRATORY TRACT INFECTION; META ANALYSIS (TOPIC); OUTCOME ASSESSMENT; PATIENT SELECTION; PILOT STUDY; PRIORITY JOURNAL; PUBLIC HEALTH; RANDOMIZED CONTROLLED TRIAL (TOPIC); RESEARCH ETHICS; RESPIRATORY SYNCYTIAL PNEUMOVIRUS; SAMPLE SIZE; STUDY DESIGN; VIRAL BRONCHIOLITIS;

HUMANS; PRACTICE GUIDELINES AS TOPIC; RANDOMIZED CONTROLLED TRIALS AS TOPIC; REPRODUCIBILITY OF RESULTS; SAMPLE SIZE;

EID: 84861862717     PISSN: 00314005     EISSN: 10984275     Source Type: Journal    
DOI: 10.1542/peds.2012-0055G     Document Type: Conference Paper

References (40)

1. Charles P, Giraudeau B, Dechartres A, Baron G, Ravaud P. Reporting of sample size calculation in randomised controlled trials: review. BMJ. 2009;338:b1732
2. Vickers AJ. Underpowering in randomized trials reporting a sample size calculation. J Clin Epidemiol. 2003;56(8):717-720 (Pubitemid 37040806)
3. van der Lee JH, Tanck MW, Wesseling J, Offringa M. Pitfalls in the design and analysis of paediatric clinical trials: a case of a 'failed' multi-centre study, and potential solutions. Acta Paediatr. 2009;98 (2):385-391
4. Lachin JM. Introduction to sample size determination and power analysis for clinical trials. Control Clin Trials. 1981;2(2):93-113 (Pubitemid 11006595)
5. Freedman LS. Tables of the number of patients required in clinical trials using the logrank test. Stat Med. 1982;1(2):121-129 (Pubitemid 13146129)
6. Donner A. Approaches to sample size estimation in the design of clinical trials-a review. Stat Med. 1984;3(3):199-214 (Pubitemid 14037609)
7. Cohen J. A power primer. Psychol Bull. 1992; 112(1):155-159
8. Julious SA. Sample sizes for clinical trials with normal data. Stat Med. 2004;23(12): 1921-1986
9. Dann RS, Koch GG. Methods for one-sided testing of the difference between proportions and sample size considerations related to non-inferiority clinical trials. Pharm Stat. 2008;7(2):130-141 (Pubitemid 351943641)
10. Friedman LM, Furberg CD, DeMets DL. Fundamentals of Clinical Trials. 4th ed. New York, NY: Springer; 2010
11. Piantadosi S. Clinical Trials. A Methodological Perspective. New York, NY: John Wiley & Sons; 1997
12. Clarke M, Hopewell S, Chalmers I. Clinical trials should begin and end with systematic reviews of relevant evidence: 12 years and waiting. Lancet. 2010;376(9734): 20-21
13. Sutton AJ, Cooper NJ, Jones DR, Lambert PC, Thompson JR, Abrams KR. Evidence-based sample size calculations based upon updated meta-analysis. Stat Med. 2007;26(12):2479-2500 (Pubitemid 46784206)
14. Whitehead A. Meta-analysis of Controlled Clinical Trials. Chichester, West Sussex, United Kingdom: Wiley; 2002
15. Cochrane Collaboration. Review Manager (RevMan). Available at: www.ims.cochrane. org/revman/download. Accessed April 28, 2012
16. Julious SA, Owen RJ. Sample size calculations for clinical studies allowing for uncertainty about the variance. Pharm Stat. 2006;5(1):29-37 (Pubitemid 43380266)
17. Henderson-Smart DJ, Steer P. Methylxanthine treatment for apnea in preterm infants. Cochrane Database Syst Rev. 2001;(3): CD000140
18. Schmidt B, Roberts RS, Davis P, et al Caffeine for Apnea of Prematurity Trial Group. Longterm effects of caffeine therapy for apnea of prematurity. N Engl J Med. 2007;357(19): 1893-1902 (Pubitemid 350074685)
19. Pillai AK, Sharma KK, Gupta YK, Bakhshi S. Anti-emetic effect of ginger powder versus placebo as an add-on therapy in children and young adults receiving high emetogenic chemotherapy. Pediatr Blood Cancer. 2011;56(2):234-238
20. Whitehead J, Valdés-Márquez E, Johnson P, Graham G. Bayesian sample size for exploratory clinical trials incorporating historical data. Stat Med. 2008;27(13): 2307-2327 (Pubitemid 351802896)
21. Goodman SN, Sladky JT. A Bayesian approach to randomized controlled trials in children utilizing information from adults: the case of Guillain-Barré syndrome. Clin Trials. 2005;2(4):305-310, discussion 364-378
22. Schoenfeld DA, Hui Zheng, Finkelstein DM. Bayesian design using adult data to augment pediatric trials. Clin Trials. 2009;6(4): 297-304
23. Stanojevic S, Wade A, Stocks J. Reference values for lung function: past, present and future. Eur Respir J. 2010;36(1):12-19
24. Wittes J, Brittain E. The role of internal pilot studies in increasing the efficiency of clinical trials. Stat Med. 1990;9(1-2):65-71; discussion 71-72
25. Freidlin B, Korn EL. Release of data from an ongoing randomized clinical trial for sample size adjustment or planning. Stat Med. 2007;26(22):4074-4082 (Pubitemid 47399341)
26. Kieser M, Friede T. Re-calculating the sample size in internal pilot study designs with control of the type I error rate. Stat Med. 2000;19(7):901-911 (Pubitemid 30185303)
27. Proschan MA, Liu Q, Hunsberger S. Practical midcourse sample size modification in clinical trials. Control Clin Trials. 2003;24 (1):4-15 (Pubitemid 36135942)
28. Gould AL, Shih WJ. Sample size reestimation without unblinding for normally distributed outcomes with unknown variance. Comm Statist Theory Methods. 1992; 21(10):2833-2853
29. Committee for Medicinal Products for Human Use. Guideline on Clinical Trials in Small Populations. London, United Kingdom: European Medicines Agency; 2006
30. Evans, Jr, CH and Ildstad ST, eds. Committee on Strategies for Small-Number-Participant. Small Clinical Trials: Issues and Challenges. Washington, DC: National Academy Press; 2001
31. Senn S. Cross-over Trials in Clinical Research. 2nd ed. Chichester, West Sussex, United Kingdom: Wiley; 2002
32. Winkens B, Schouten HJ, van Breukelen GJ, Berger MP. Optimal number of repeated measures and group sizes in clinical trials with linearly divergent treatment effects. Contemp Clin Trials. 2006;27(1):57-69 (Pubitemid 41758439)
33. Haybittle JL. Repeated assessment of results in clinical trials of cancer treatment. Br J Radiol. 1971;44(526):793-797
34. Peto R, Pike MC, Armitage P, et al. Design and analysis of randomized clinical trials requiring prolonged observation of each patient. I. Introduction and design. Br J Cancer. 1976;34(6):585-612 (Pubitemid 8004970)
35. O'Brien PC, Fleming TR. A multiple testing procedure for clinical trials. Biometrics. 1979;35(3):549-556 (Pubitemid 10242750)
36. DeMets DL, Lan KK. Interim analysis: the alpha spending function approach. Stat Med. 1994;13(13-14):1341-1352; discussion 1353-1356
37. Ghersi D, Berlin J, Askie L. Chapter 19: Prospective meta-analysis. In: Higgins J, Green S, eds. Cochrane Handbook for Systematic Reviews of Interventions Version 5.1.0 (updated March 2011). The Cochrane Collaboration; 2011. Available at: www.cochranehandbook. org. Accessed April 28, 2012
38. Askie LM, Baur LA, Campbell K, et al EPOCH Collaboration Group. The Early Prevention of Obesity in CHildren (EPOCH) Collaboration-an individual patient data prospective meta-analysis. BMC Public Health. 2010;10:728
39. Bauer P, Köhne K. Evaluation of experiments with adaptive interim analyses. Biometrics. 1994;50(4):1029-1041
40. Bauer P, Brannath W. The advantages and disadvantages of adaptive designs for clinical trials. Drug Discov Today. 2004;9(8): 351-357 (Pubitemid 38479690)