Evidence-Based Practice in Healthcare: Key Concepts and Applications

The Culture of Health Care Evidence-Based Practice Lecture e This material (Comp 2 Unit 5) was developed by Oregon Health & Science University, funded by the Department of Health and Human Services, Office of the National Coordinator for Health Information Technology under Award Number IU24OC000015. This material was updated in 2016 by Bellevue College under Award Number 90WT0002. This work is licensed under the Creative Commons Attribution-NonCommercial-ShareAlike 4.0 International License. To view a copy of this license, visit http://creativecommons.org/licenses/by-nc-sa/4.0/.

Evidence-Based Practice Learning Objectives Define the key tenets of evidence-based medicine (EBM) and its role in the culture of health care (Lectures a, b). Construct answerable clinical questions and critically appraise evidence answering them (Lecture b). Explain how EBM can be applied to intervention studies, including the phrasing of answerable questions, finding evidence to answer them, and applying them to given clinical situations (Lecture c). Describe how EBM can be applied to key clinical questions of diagnosis, harm, and prognosis (Lectures d, e). Discuss the benefits and limitations to summarizing evidence (Lecture f). Describe how EBM is used in clinical settings through clinical practice guidelines and decision analysis (Lecture g). 3

Using EBM to Assess Questions about Harm or Etiology Question is not whether someone exposed to agent gets ill, but whether those with illness have higher rate or amount of exposure Ideally assessed by randomized controlled trial, but RCT may be impractical or unethical Next best evidence comes from observational studies, which have limitations 4

Examples of Questions to Answer about Harm Do silicone breast implants cause autoimmune diseases, such as lupus? (Gabriel et al., 1994; Vase, 2013) Women with silicone breast implants developed connective tissue diseases and arthritis but at no higher rate than those without them Do anti-obesity drugs (e.g., fenfluramine and phentermine, also known as fen-phen) cause heart valve abnormalities? (Gardin et al., 2000; Andrejak, 2013) Those who used these drugs developed certain heart valve abnormalities at a higher rate than those who did not 5

Hierarchy of Evidence for Harm Randomized controlled trial Cohort study Case-control study Case series/report 6

Evidence and Its Limits Randomized controlled trial Ideal, but often cannot be done or would be unethical to do so Cohort study Prospective study without randomization Particularly useful when poor outcomes are rare and huge sample size would be required e.g., upper gastrointestinal hemorrhage with NSAIDs Problematic when groups are dissimilar e.g., people who take NSAIDS may be sicker than or otherwise different from those who do not 7

Evidence and Its Limits Continued Case-control study Most common form of observational study Retrospectively identify cases of diseases and match to otherwise similar controls, looking to see if different rate or amount of exposure Useful when condition is very rare or has long development time o Classic case was demonstration that DES causes vaginal cancer (reviewed in Swan, 2000) 8

Evidence and Its Limits Continued 2 Case-control study continued Problem is when controls create spurious association, e.g., o Coffee drinking associated with miscarriages o 2016 NIH article says yes, Slate magazine article disputes findings o pancreatic cancer (MacMahon et al., 1981), but controls were patients with other GI diseases whose symptoms were exacerbated by coffee (so they drank less) o Differences were not present when other appropriate controls were used (Zheng et al., 1993) 9

Evidence and Its Limits Continued 3 Case series/report o No comparison group o Famous example was Bendectin for nausea in pregnancy, where adverse publicity led to removal from market of safe and effective treatment Was combination of two agents, both of which were effective and neither of which were harmful (Magee, Mazzotta, & Koren, 2002; Hale, 2012) Re-introduced use of ingredients in a delayed-release combination pill after US FDA approval in 2013 (Nuangchamnong, 2014) 10

Pure Prognosis Studies Are Rare Prognosis is natural history of disease But very little history is natural in modern era with our abundance of diagnostic tests, interventions, harmful agents, and so on Many studies measure prognosis after a test or intervention 11

Prognosis Usually Measured by a Survival Curve 5.5 Chart: Survival Curve (Adapted from Dunn, 2002) 12

Example Studies of Prognosis Extremely preterm birth (Marlow, Wolke, & Bracewell, 2005) Followed cohort of 241 children from UK and Ireland born at 25 or fewer weeks gestation Compared with 160 classmates born at full term 41% of preterm children had serious impairment on cognitive assessment compared with 1.3% in control group Untreated early, localized prostate cancer (Johansson et al., 2004; also see Yao, 2010; Gulati,. 2011) 223 men followed from 1977 to 1984 17% developed generalized disease 16% died of disease 13

Evidence-Based Practice Summary Lecture e Questions about harm assess whether exposure to some natural or manmade agent causes disease and is usually answered with a case-control study Questions about prognosis tell us the natural course of disease 14

Evidence-Based Practice References Lecture e References Al-Dakkak, I. (2011). Tea, coffee and oral cancer risk. Evidence-Based Dentistry, 12, 23 24. Retrieved from http://www.nature.com/ebd/journal/v12/n1/pdf/6400780a.pdf Alsop, J., Scott, M., & Archey, W. (2016). The mixed randomized trial: combining randomized, pragmatic and observational clinical trial designs. Journal of Comparative Effectiveness Research, 5(6), 569-579. Retrieved from http://www.futuremedicine.com/doi/abs/10.2217/cer- 2016-0034 American College of Obstetrics and Gynecologists. (2010). Moderate caffeine consumption during pregnancy. (Committee Opinion #462), Obstetrics & Gynecology. 116.2: 467-468. Andrejak, M., & Tribouilloy, C. (2013). Drug-induced valvular heart disease: an update. Archives of Cardiovascular Diseases, 106(5), 333-339. Anonymous. (2015). Beware spurious correlations. Harvard Business Review. June. Retrieved from https://hbr.org/2015/06/beware-spurious-correlations Faraoni, D., & Schaefer, S. T. (2016). Randomized controlled trials vs. observational studies: why not just live together?. BMC anesthesiology, 16(1), 102. Retrieved from https://bmcanesthesiol.biomedcentral.com/articles/10.1186/s12871-016-0265-3 Ferreira, J. C., & Patino, C. M. (2016). Choosing wisely between randomized controlled trials and observational designs in studies about interventions. Jornal Brasileiro de Pneumologia, 42(3), 165-165. Retrieved from http://www.scielo.br/scielo.php?pid=S1806- 37132016000300165&script=sci_arttext Gulati, R. et als. (2011). What if I don t treat my PSA-detected prostate cancer? Answers from three natural history models. Cancer Epidemiology Biomarkers Prevention. 20(5):740-750. Retrieved from http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3091266/pdf/nihms262673.pdf 15

Evidence-Based Practice References Lecture e Continued References Gabriel, S., O Fallon, W., Kurland, L., Beard, C., Woods, J., & Melton, L. (1994). Risk of connective- tissue diseases and other disorders after breast implantation. New England Journal of Medicine, 330, 1697 1702. Gardin, J., Schumacher, D., Constantine, G., Davis, K., Leung, C., & Reid, C. (2000). Valvular abnormalities and cardiovascular status following exposure to dexfenfluramine or phentermine/fenfluramine. Journal of the American Medical Association, 283, 1703 1709. Hale, R. W., & Niebyl, J. (2012). Bendectin: how a safe and effective drug was removed from the market by our legal system. ACOG Clinical Review, 17(5), 25-27. Johansson, J., Andren, O., Andersson, S., Dickman, P., Holmberg, L., Magnuson, A., & Adami, H. (2004). Natural history of early, localized prostate cancer. Journal of the American Medical Association, 291, 2713 2719. MacMahon, B., Yen, S., Trichopoulos, D., Warren, K., & Nardi, G. (1981). Coffee and cancer of the pancreas. New England Journal of Medicine, 304, 630 633. Magee, L., Mazzotta, P., & Koren, G. (2002). Evidence-based view of safety and effectiveness of pharmacologic therapy for nausea and vomiting of pregnancy (NVP). American Journal of Obstetrics and Gynecology, 186, S256 S261. Marlow, N., Wolke, D., & Bracewell, M. (2005). Neurologic and developmental disability at six years of age after extremely preterm birth. New England Journal of Medicine, 352, 9 19. Nuangchamnong, N., Niebyl, J. (2014). Doxylamine succinate pyridoxine hydrochloride (Diclegis) for the management of nausea and vomiting in pregnancy: an overview. International Journal of Women s Health, 6: 401 409. 16

Evidence-Based Practice References Lecture e Continued 2 References Swan, S. (2000). Intrauterine exposure to diethylstilbestrol: Long-term effects in humans. Acta Pathologica, Microbiologica et Immunologica Scandinavica, 108, 793 804. Vase, M. ., Friis, S., Bautz, A., Bendix, K., S rensen, H. T., & d'Amore, F. (2013). Breast implants and anaplastic large-cell lymphoma: a Danish population-based cohort study. Cancer Epidemiology Biomarkers & Prevention, 22(11), 2126-2129. Retrieved from http://cebp.aacrjournals.org/content/22/11/2126.full.pdf+html Weng, Xiaoping, Ph.D., Odoili, R. MSPH, Li De-Kun, MD, PhD. Kaiser Permanente Division of Research. (2008). Maternal caffeine consumption during pregnancy and the risk of miscarriage: a prospective cohort study. American Journal of Obstetrics and Gynecology, 198 (3), 279.e1- 279.e8. Retrieved from http://ac.els-cdn.com/S000293780702025X/1-s2.0-S000293780702025X- main.pdf?_tid=806fef54-0b1a-11e6-8391- 00000aacb360&acdnat=1461612093_f3e8a62dfeb80d8bad4dce4c14d202e2 Yadav, D., & Lowenfels, A. B. (2013). The epidemiology of pancreatitis and pancreatic cancer. Gastroenterology, 144(6), 1252-1261. Retrieved from http://www.gastrojournal.org/article/S0016-5085(13)00168-6/pdf Yao, S. & Lu-You, G. (2010). Diagnosis of localized, screen-detected, prostate cancer Crisis or opportunity? Journal of the National Cancer Institute. 102(13): 919 920. Retrieved from http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2897880/pdf/djq219.pdf Zheng, W., et al. (1993). A cohort study of smoking, alcohol consumption, and dietary factors for pancreatic cancer (United States). Cancer Causes & Control, 4, 477 482.

Evidence-Based Practice References Lecture e Continued 3 Charts, Tables, Figures 5.5 Chart: Survival Curve. Adapted from Dunn, S. (2002). Survival curves: The Basics. CancerGuide. Retrieved from http://cancerguide.org/scurve_basic.html 18

The Culture of Health Care Evidence-Based Practice Lecture e This material was developed by Oregon Health & Science University, funded by the Department of Health and Human Services, Office of the National Coordinator for Health Information Technology under Award Number IU24OC000015. This material was updated in 2016 by Bellevue College under Award Number 90WT0002. 19