Skip to main content

The role of randomized controlled trials in evidence-based urology

Abstract

Purpose

To review the current and future role of randomized controlled trials (RCTs) in urology.

Methods

A review of the urological literature was performed to assess the current role of RCTs in urology. These findings were put in context of current practice, and methodologic limitations of observational study design were discussed.

Results

Several RCTs have improved treatment of urology patients. However, overall, few studies in the urologic literature convey high-level evidence. RCTs represent the only study design that can assess causality, thus are the only means to significantly advance knowledge about therapy and improve patient care. Increased incidence and improved quality of RCTs may be achieved through education, community outreach, and better access to research infrastructure.

Conclusions

RCTs have significantly improved urologic practice. RCTs are superior to observational studies because of decreased risk of bias and confounding. To further advance clinical practice, clinical trials must play a predominant role and should be embraced by the urology community.

This is a preview of subscription content, access via your institution.

References

  1. Blom JH, van Poppel H, Marechal JM, Jacqmin D, Schroder FH, de Prijck L, Sylvester R, EORTC Genitourinary Tract Cancer Group (2009) Radical nephrectomy with and without lymph-node dissection: final results of European Organization for Research and Treatment of Cancer (EORTC) randomized phase 3 trial 30881. Eur Urol 55:28–34

    PubMed  Article  Google Scholar 

  2. Borawski KM, Norris RD, Fesperman SF, Vieweg J, Preminger GM, Dahm P (2007) Levels of evidence in the urological literature. J Urol 178:1429–1433

    PubMed  Article  Google Scholar 

  3. Welk B, Afshar K, MacNeily AE (2006) Randomized controlled trials in pediatric urology: room for improvement. J Urol 176:306–309 Discussion 309-310

    PubMed  Article  Google Scholar 

  4. Breau RH, Karnes RJ, Jacobson DJ, McGree ME, Jacobsen SJ, Nehra A, Lieber MM, St Sauver JL (2010) The association between statin use and the diagnosis of prostate cancer in a population based cohort. J Urol 184:494–499

    PubMed  Article  Google Scholar 

  5. Flick ED, Habel LA, Chan KA, Van Den Eeden SK, Quinn VP, Haque R, Orav EJ, Seeger JD, Sadler MC, Quesenberry CP Jr, Sternfeld B, Jacobsen SJ, Whitmer RA, Caan BJ (2007) Statin use and risk of prostate cancer in the California men’s health study cohort. Cancer Epidemiol Biomarkers Prev 16:2218–2225

    PubMed  Article  CAS  Google Scholar 

  6. Andriole GL, Bostwick DG, Brawley OW, Gomella LG, Marberger M, Montorsi F, Pettaway CA, Tammela TL, Teloken C, Tindall DJ, Somerville MC, Wilson TH, Fowler IL, Rittmaster RS, REDUCE Study Group (2010) Effect of dutasteride on the risk of prostate cancer. N Engl J Med 362:1192–1202

    PubMed  Article  CAS  Google Scholar 

  7. Thompson IM, Goodman PJ, Tangen CM, Lucia MS, Miller GJ, Ford LG, Lieber MM, Cespedes RD, Atkins JN, Lippman SM, Carlin SM, Ryan A, Szczepanek CM, Crowley JJ, Coltman CA Jr (2003) The influence of finasteride on the development of prostate cancer. N Engl J Med 349:215–224

    PubMed  Article  CAS  Google Scholar 

  8. McConnell JD, Roehrborn CG, Bautista OM, Andriole GL Jr, Dixon CM, Kusek JW, Lepor H, McVary KT, Nyberg LM Jr, Clarke HS, Crawford ED, Diokno A, Foley JP, Foster HE, Jacobs SC, Kaplan SA, Kreder KJ, Lieber MM, Lucia MS, Miller GJ, Menon M, Milam DF, Ramsdell JW, Schenkman NS, Slawin KM, Smith JA, Medical Therapy of Prostatic Symptoms (MTOPS) Research Group (2003) The long-term effect of doxazosin, finasteride, and combination therapy on the clinical progression of benign prostatic hyperplasia. N Engl J Med 349:2387–2398

    PubMed  Article  CAS  Google Scholar 

  9. Thompson IM, Pauler DK, Goodman PJ, Tangen CM, Lucia MS, Parnes HL, Minasian LM, Ford LG, Lippman SM, Crawford ED, Crowley JJ, Coltman CA Jr (2004) Prevalence of prostate cancer among men with a prostate-specific antigen level < or =4.0 ng per milliliter. N Engl J Med 350:2239–2246

    PubMed  Article  CAS  Google Scholar 

  10. Darouiche RO, Wall MJ Jr, Itani KM, Otterson MF, Webb AL, Carrick MM, Miller HJ, Awad SS, Crosby CT, Mosier MC, Alsharif A, Berger DH (2010) Chlorhexidine-alcohol versus povidone-iodine for surgical-site antisepsis. N Engl J Med 362:18–26

    PubMed  Article  CAS  Google Scholar 

  11. Dellabella M, Milanese G, Muzzonigro G (2003) Efficacy of tamsulosin in the medical management of juxtavesical ureteral stones. J Urol 170:2202–2205

    PubMed  Article  CAS  Google Scholar 

  12. Freedman B (1987) Equipoise and the ethics of clinical research. N Engl J Med 317:141–145

    PubMed  Article  CAS  Google Scholar 

  13. Moher D, Hopewell S, Schulz KF, Montori V, Gotzsche PC, Devereaux PJ, Elbourne D, Egger M, Altman DG, Consolidated Standards of Reporting Trials Group (2010) CONSORT 2010 explanation and elaboration: updated guidelines for reporting parallel group randomised trials. J Clin Epidemiol 63:e1–e37

    PubMed  Article  Google Scholar 

  14. Andriole GL, Crawford ED, Grubb RL III, Buys SS, Chia D, Church TR, Fouad MN, Gelmann EP, Kvale PA, Reding DJ, Weissfeld JL, Yokochi LA, O’Brien B, Clapp JD, Rathmell JM, Riley TL, Hayes RB, Kramer BS, Izmirlian G, Miller AB, Pinsky PF, Prorok PC, Gohagan JK, Berg CD, PLCO Project Team (2009) Mortality results from a randomized prostate-cancer screening trial. N Engl J Med 360:1310–1319

    PubMed  Article  CAS  Google Scholar 

  15. Schulz KF, Grimes DA (2002) Blinding in randomised trials: hiding who got what. Lancet 359:696–700

    PubMed  Article  Google Scholar 

  16. Lavallee LT, Dahm P, Breau RH (2010) Evidence-based urology in practice: composite endpoints. BJU Int 106:610–612

    PubMed  Article  Google Scholar 

  17. Bucher HC, Guyatt GH, Cook DJ, Holbrook A, McAlister FA (1999) Users’ guides to the medical literature: XIX. Applying clinical trial results. A. How to use an article measuring the effect of an intervention on surrogate end points. Evidence-based medicine working group. JAMA 282:771–778

    PubMed  Article  CAS  Google Scholar 

  18. Hadley J, Yabroff KR, Barrett MJ, Penson DF, Saigal CS, Potosky AL (2010) Comparative effectiveness of prostate cancer treatments: evaluating statistical adjustments for confounding in observational data. J Natl Cancer Inst 102:1780–1793

    PubMed  Article  Google Scholar 

  19. Yoshizawa K, Willett WC, Morris SJ, Stampfer MJ, Spiegelman D, Rimm EB, Giovannucci E (1998) Study of prediagnostic selenium level in toenails and the risk of advanced prostate cancer. J Natl Cancer Inst 90:1219–1224

    PubMed  Article  CAS  Google Scholar 

  20. Nomura AM, Lee J, Stemmermann GN, Combs GF Jr (2000) Serum selenium and subsequent risk of prostate cancer. Cancer Epidemiol Biomarkers Prev 9:883–887

    PubMed  CAS  Google Scholar 

  21. Duffield-Lillico AJ, Dalkin BL, Reid ME, Turnbull BW, Slate EH, Jacobs ET, Marshall JR, Clark LC, Nutritional Prevention of Cancer Study Group (2003) Selenium supplementation, baseline plasma selenium status and incidence of prostate cancer: an analysis of the complete treatment period of the nutritional prevention of cancer trial. BJU Int 91:608–612

    PubMed  Article  CAS  Google Scholar 

  22. Heinonen OP, Albanes D, Virtamo J, Taylor PR, Huttunen JK, Hartman AM, Haapakoski J, Malila N, Rautalahti M, Ripatti S, Maenpaa H, Teerenhovi L, Koss L, Virolainen M, Edwards BK (1998) Prostate cancer and supplementation with alpha-tocopherol and beta-carotene: incidence and mortality in a controlled trial. J Natl Cancer Inst 90:440–446

    PubMed  Article  CAS  Google Scholar 

  23. Lippman SM, Klein EA, Goodman PJ, Lucia MS, Thompson IM, Ford LG, Parnes HL, Minasian LM, Gaziano JM, Hartline JA, Parsons JK, Bearden JD III, Crawford ED, Goodman GE, Claudio J, Winquist E, Cook ED, Karp DD, Walther P, Lieber MM, Kristal AR, Darke AK, Arnold KB, Ganz PA, Santella RM, Albanes D, Taylor PR, Probstfield JL, Jagpal TJ, Crowley JJ, Meyskens FL Jr, Baker LH, Coltman CA Jr (2009) Effect of selenium and vitamin E on risk of prostate cancer and other cancers: the selenium and vitamin E cancer prevention trial (SELECT). JAMA 301:39–51

    PubMed  Article  CAS  Google Scholar 

  24. Dahm P, Preminger GM, Scales CD Jr, Fesperman SF, Yeung LL, Cohen MS (2009) Evidence-based medicine training in residency: a survey of urology programme directors. BJU Int 103:290–293

    PubMed  Article  Google Scholar 

  25. Melton LJ III (1996) History of the rochester epidemiology project. Mayo Clin Proc 71:266–274

    PubMed  Article  Google Scholar 

  26. Scales CD Jr, Voils CI, Fesperman SF, Sur RL, Kubler H, Preminger GM, Dahm P (2008) Barriers to the practice of evidence-based urology. J Urol 179:2345–2349 discussion 2349-50

    PubMed  Article  Google Scholar 

  27. Schaeffer AJ (2009) NIH urological funding for the last 5 years: the good and the bad. AUANews 14:16–19

    Google Scholar 

  28. Lent V, Langenbach A (1996) A retrospective quality analysis of 102 randomized trials in four leading urological journals from 1984–1989. Urol Res 24:119–122

    PubMed  Article  CAS  Google Scholar 

  29. Scales CD Jr, Norris RD, Keitz SA, Peterson BL, Preminger GM, Vieweg J, Dahm P (2007) A critical assessment of the quality of reporting of randomized, controlled trials in the urology literature. J Urol 177:1090–1094 discussion 1094-5

    PubMed  Article  Google Scholar 

  30. Agha R, Cooper D, Muir G (2007) The reporting quality of randomised controlled trials in surgery: a systematic review. Int J Surg 5:413–422

    PubMed  Article  Google Scholar 

Download references

Conflict of interest

The authors declare that they have no conflict of interest.

Author information

Affiliations

Authors

Corresponding author

Correspondence to Rodney H. Breau.

Rights and permissions

Reprints and Permissions

About this article

Cite this article

Lavallée, L.T., Fergusson, D. & Breau, R.H. The role of randomized controlled trials in evidence-based urology. World J Urol 29, 257–263 (2011). https://doi.org/10.1007/s00345-011-0646-7

Download citation

  • Received:

  • Accepted:

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1007/s00345-011-0646-7

Keywords

  • Evidence-based medicine
  • Randomized controlled trial
  • Urology