TUMAČENJE REZULTATA KLINIČKIH RANDOMIZOVANIH STUDIJA
Sažetak
Cilj ovog preglednog rada je da kroz primere objasni kako izabrati, računati i tumačiti mere efekta u kliničkim randomizovanim studijama. Kliničke randomizovane studije (engl. Randomized Control Trials, RCTs) pripadaju grupi eksperimentalnih studija i sprovode se nad obolelim osobama. U ovim studijama ispitanici se metodom randomizacije raspoređuju u eksperimentalnu grupu, koja dobija ispitivano sredstvo, i kontrolnu, koja prima placebo ili neko do tada korišćeno sredstvo. Rezultati RCTs mogu se prezentovati kroz relativne i apsolutne mere efekta. Relativne mere efekta su relativni rizik (engl. Relative Risk, RR) i relativno smanjenje rizika (engl. Relative Risk Reduction, RRR), a apsolutne mere efekta su apsolutno smanjenje rizika (engl. Absolute Risk Reduction, ARR) i broj pacijenata koje treba lečiti (engl. Number Needed to Treat, NNT). Najjači dokaz između uzroka i posledice dobija se u ovim studijama, ali se ove studije retko izvode zbog svoje cene. Mali broj ispitanika u ovim studijama, može se prevazići korišćenjem meta-analize i multicentričnih studija.
Reference
Gamulin S. Klinička istraživanja – klinička epidemiologija. Zagreb: Medicinska naklada, 2015. 144 p.
European Commission. Clinical trials – Regulation (EU) No 536/2014 [Internet]. [cited August 2023]. Avilable at: https://www.health.ec.europa.eu
Etminan M, Wright JM, Carleton BC. Evidence-based pharmacotherapy: review of basic concepts and applications in clinical practice. Ann Pharmacother 1998;32(11):1193-200. doi: 10.1345/aph.17333.
Higgins JPT, Li T, Deeks JJ. Chapter 6: Choosing effect measures and computing estimates of effects. In: Higgins JPT, Thomas J, Chandler J, Cumpston M, Li T, Page MJ, Welch VA (editors). Cochrane Handbook for Systematic Reviews of Interventions version 6.3 (updated February 2022). Cochrane, 2022. Available at: www.training.cochrane.org/handbook
The SOLVD Investigators. Effect of enalapril on survival in patients with reduced ejection fraction and congestive heart failure. N Engl J Med 1991;325(5):293-302. doi:10.1056/NEJM199108013250501.
Darzi AJ, Busse JW, Phillips M, Wykoff CC, Guymer RH, Thabane L, et al. Interpreting results from randomized controlled trials: What measures to focus on in clinical practice. Eye (Lond) 2023; doi: 10.1038/s41433-023-02454-7. Online ahead of print.
Andrade C. Understanding realtive risk, odds ratio, and related terms: as simple as it can get. J Clin Psychiatry 2015; 76(7):e857-61. doi: 10.4088/JCP.15f10150.
Citrome L, Ketter TA. When does a difference make a difference? Interpretation of number needed to treat, number needed to harm, and likelihood to be helped or harmed. Int J Clin Pract 2013;67(5): 407-11. doi: 10.4088/JCP.15f10150.
Cookson J, Gilaberte I, Desaiah D, Kajdasz DK. Treatment benefits of duloxetine in major depressive disorder as assessed by number needed to treat. Int Clin Psychopharmacol 2006;21(5):267-73. doi:10.1097/00004850-200609000-00004.
Agarwal A, Johnston BC, Vernooij RWM, Carrasco-Labra A, Brignardello-Petersen R, Neumann I, et al. Authors seldom report the most patient-important outcomes and absolute effect measures in systematic review abstracts. J Clin Epidemiol 2017;81:3-12. doi: 10.1016/j.jclinepi.2016.08.004.
Andersen LW. Absolute vs. Relative effects-implications for subgroup analyses. Trials 2021; 22(1):50. doi:10.1186/s13063-020-05005-7.
Sormani MP, Bruzzi P. Estimating a treatment effect: choosing between relative and absolute measures. Mult Scler 2017;23(2):197-200.
Štimac D, Lackovic A, Poropat G. Važnost provođenja randomiziranih kliničkih istraživanja. Medicina Fluminensis 2017; 53(4):448-53. doi: 10.21860/medflum2017_187364.
Saturni S, Bellini F, Braido F, Paggiaro P, Sanduzzi A, Scichilone N, et al. Randomized controlled trials and real-life studies. Approaches and methodologies: a clinical point of view. Pulm Pharmacol Ther 2014;27(2):129-38. doi:10.1016/j.pupt.2014.01.005.
Hariton E, Locascio J. Randomised controlled trials – the gold standard for effectiveness research. BJOG 2018; 125(13):1716. doi: 10.1111/1471-0528.15199.
Speich B, von Neiderhäusern B, Schur N, Hemkens LG, Fürst T, Bhatnagar N, et al. Systematic review on costs and resource use of randomized clinical trials shows a lack of transparent and comprehensive data. J Clin Epidemiol 2018:96:1-11. doi: 10.1016/j.jclinepi.2017.12.018