Advertisement

Pitfalls in the Design, Analysis, Presentation, and Interpretation of Randomized Clinical Trials

  • Richard Stephens
Chapter
Part of the Medical Radiology book series (MEDRAD)

Abstract

Randomised clinical trials are the gold standard tool for testing new treatments, but need to confirm to certain basic guidelines in order to be considered reliable. Good trial design, analysis, presentation and interpretation and an awareness of the common pitfalls are likely to maximise the relevance and impact of the trial.

Keywords

Prophylactic Cranial Irradiation Multiple Statistical Testing Ineligible Patient Prognostic Factor Analysis Prophylactic Cranial Irradiation Group 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.

References

  1. Clarke M, Chalmers I (1998) Discussion sections in reports of controlled trials published in general medical journals: Islands in search of continents? JAMA 280:280–282PubMedCrossRefGoogle Scholar
  2. Clarke M, Alderson P, Chalmers I (2002) Discussion sections in reports of controlled trial published in general medical journals. JAMA 287:2799–2801PubMedCrossRefGoogle Scholar
  3. Fossella FV, DeVore R, Kerr RN, Crawford J, Natale RR et al (2000) Randomized phase III trial of docetaxel versus vinorelbine or ifosfamide in patients with advanced non-small cell lung cancer previously treated with platinum-containing chemotherapy regimens. J Clin Oncol 18:2354–2362PubMedGoogle Scholar
  4. Fukuoka M, Yano S, Giaccone G, Tamura T, Nagagawa K et al (2003) Multi-institutional randomized phase II trial of gefitinib for previously treated patients with advanced non-small cell lung cancer. J Clin Oncol 21:2237–2246PubMedCrossRefGoogle Scholar
  5. Gatzemeier U, von Pawel J, Gottfried M, ten Velde GPM, Mattson K et al (2000) Phase III comparative study of high dose cisplatin versus a combination of paclitaxel and cisplatin in patients with advanced non-small cell lung cancer. J Clin Oncol 18:3390–3399PubMedGoogle Scholar
  6. Georgoulias V, Papadakis E, Alexopoulos A, Tsiafaki X, Rapti A et al (2001) Platinum-based and non-platinum-based chemotherapy in advanced non-small cell lung cancer: a randomised multicentre trial. Lancet 357:1478–1484PubMedCrossRefGoogle Scholar
  7. Gore EM, Bae K, Wong SJ, Sun A, Bonner JA et al (2011) Phase III Comparison of prophylactic cranial irradiation versus observation in patients with locally advanced non-small-cell lung cancer: primary analysis of radiation therapy oncology group study RTOG 0214. J Clin Oncol 29:272–278PubMedCrossRefGoogle Scholar
  8. Kelly K, Crowley J, Bunn PA Jr, Presant CA, Grevstad PK et al (2001) Randomized phase III trial of paclitaxel plus carboplatin versus vinorelbine plus cisplatin in the treatment of patients with advanced non-small cell lung cancer: a southwest oncology group trial. J Clin Oncol 19:3210–3218PubMedGoogle Scholar
  9. Langendijk H, de Jong J, Tjwa M, Muller M, ten Velde G et al (2001) External irradiation versus external irradiation plus endobronchial brachytherapy in inoperable non-small cell lung cancer: a prospective randomized study. Rad Oncol 58:257–268CrossRefGoogle Scholar
  10. Lee JJ (2011) Demystify statistical significance—time to move on from the p value to Bayesian analysis. J Natl Cancer 103:2–3CrossRefGoogle Scholar
  11. Non-Small Cell Lung Cancer Collaborative Group (1995) Chemotherapy in non-small cell lung cancer: a meta-analysis using updated data on individual patients from 52 randomised clinical trials. BMJ 311:899–909CrossRefGoogle Scholar
  12. Pujol J-L, Daures J-P, Riviere A, Quoix E, Westell V et al (2001) Etoposide plus cisplatin with or without the combination of 4′-epidoxorubicin plus cyclophosphamide in treatment of extensive small cell lung cancer: a French Federation of Cancer Institutes multicenter phase III randomized study. J Natl Cancer Inst 93:300–308PubMedCrossRefGoogle Scholar
  13. Ranson M, Davidson N, Nicolson M, Falk S, Carmichael J et al (2000) Randomized trial of paclitaxel plus supportive care versus supportive care for patients with advanced non-small cell lung cancer. J Natl Cancer Inst 92:1074–1080PubMedCrossRefGoogle Scholar
  14. Rosell R, Gomez-Condina J, Camps C, Maestre J, Padilla J et al (1994) A randomized trial comparing preoperative chemotherapy plus surgery with surgery alone in patients with non-small cell lung cancer. N Engl J Med 330:153–158PubMedCrossRefGoogle Scholar
  15. Roszkowski K, Pluzanska A, Krzakowski M, Smith AP, Saigi E et al (2000) A multicenter, randomized, phase III study of docetaxel plus best supportive care versus best supportive care in chemotherapy-naïve patients with metastatic or non-resectable localized non-small cell lung cancer (NSCLC). Lung Cancer 27:145–147PubMedCrossRefGoogle Scholar
  16. Roth JA, Fossella F, Komaki R, Ryan MB, Putnam JB et al (1994) A randomized trial comparing perioperative chemotherapy and surgery with surgery alone in resectable stage IIIa non-small cell lung cancer. J Natl Cancer Inst 86:673PubMedCrossRefGoogle Scholar
  17. Sandler AB, Nemunaitis J, Denham C, von Pawel J, Cormier Y et al (2000) Phase III trial of gemcitabine plus cisplatin versus cisplatin alone in patients with locally advanced or metastatic non-small cell lung cancer. J Clin Oncol 18:122–130PubMedGoogle Scholar
  18. Sause W, Kolesar P, Taylor IV S, Johnson D, Livingston R et al (2000) Final results of phase III trial in regionally advanced unresectable non-small cell lung cancer. Chest 117:358–364PubMedCrossRefGoogle Scholar
  19. Scagliotti GV, Fossati R, Torri V, Crino L, Giaconne G et al (2003) Randomized study of adjuvant chemotherapy for completely resected stage I, II or IIIa non-small cell lung cancer. J Natl Cancer Inst 95:1453–1461PubMedCrossRefGoogle Scholar
  20. Schiller JH, Harrington D, Belani CP, Langer C, Sandler A et al (2002) Comparison of four chemotherapy regimens for advanced non-small cell lung cancer. N Engl J Med 346:92–98PubMedCrossRefGoogle Scholar
  21. Sculier JP, Paesmans M, Lecomte J, van Cutsem O, Lafitte JJ et al (2001) A three-arm phase III randomised trial assessing, in patients with extensive disease small cell lung cancer, accelerated chemotherapy with support of haematological growth factor or oral antibiotics. Br J Cancer 85:1444–1451PubMedCrossRefGoogle Scholar
  22. Shepherd FA, Giaccone G, Seymour L, Debruyne C, Bezjak A et al (2002) Prospective, randomized, double-blind, placebo-controlled trial of marimastat after response to first-line chemotherapy in patients with small cell lung cancer: a trial of the national Cancer Institute of Canada-Clinical Trials group and the European Organization for Research and Treatment of Cancer. J Clin Oncol 20:4434–4439PubMedCrossRefGoogle Scholar
  23. Socinski MA, Schell MJ, Peterman A, Bakri K, Yates S et al (2002) Phase III trial comparing a defined duration of therapy versus continuous therapy followed by second-line therapy in advanced stage IIIb/IV non-small cell lung cancer. J Clin Oncol 20:1335–1343PubMedCrossRefGoogle Scholar
  24. Splinter TA, Sahmoud T, Festen J, van Zandwijk N, Sorenson S et al (1996) Two schedules of teniposide with or without cisplatin in advanced non-small cell lung cancer: a randomized study of the European Organization for Research and Treatment of Cancer Lung Cancer Cooperative Group. J Clin Oncol 14:127–134PubMedGoogle Scholar
  25. Sundstrom S, Bremnes RM, Kaasa S, Aasebo U, Hatlevoll R et al (2002) Cisplatin and etoposide regimen is superior to cyclophosphamide, epirubicin and vincristine regimen in small cell lung cancer: results from a randomized phase III trial with 5 years’ follow-up. J Clin Oncol 20:4665–4672PubMedCrossRefGoogle Scholar
  26. Tada H, Tsuchiya R, Ichinose Y, Koike T, Nishizawa N, Nagai K, Kato H (2004) A randomized trial comparing adjuvant chemotherapy versus surgery alone for completely resected pN2 non-small cell lung cancer (JCOG9304). Lung Cancer 43:167–173PubMedCrossRefGoogle Scholar
  27. Takada M, Fukuoka M, Kawahara M, Sugiura T, Yokohama A et al (2002) Phase III study of concurrent versus sequential thoracic radiotherapy in combination with cisplatin and etoposide for limited stage small cell lung cancer: results of the Japan Clinical Oncology Group Study 9104. J Clin Oncol 20:3054–3060PubMedCrossRefGoogle Scholar

Copyright information

© Springer-Verlag Berlin Heidelberg  2011

Authors and Affiliations

  1. 1.Clinical Trials UnitLondonUK

Personalised recommendations