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Cases in Point: Learning from Experience

INTRODUCTION

Media accounts of medical research breakthroughs are full of examples of trial and study results that make headlines because of their potential to improve patient health or even save lives—but those headlines are sometimes misleading or limited in relevance to real-world care. This cycle has caused confusion and distrust among patients and consumers of health care. At the other end of the spectrum, researchers discount the value of some methodologies used to evaluate clinical effectiveness. In part these findings reflect the constantly evolving nature of scientific inquiry; but as illustrated in this chapter, these experiences offer lessons on the improvements needed in the design and interpretation of clinical effectiveness studies.

By reviewing examples of high-profile studies and trials that evaluated the effectiveness of hormone replacement therapy, drug-eluting coronary stents, bariatric surgery, antipsychotic medications, and lung cancer screening, this chapter illustrates the range of issues facing current effectiveness research. Examples of these issues include capturing important health outcomes throughout the lifecycle of an intervention; contending with the biologic complexity of disease and disease progression and rapid evolution of devices or surgical procedures or rapid uptake and application in broader patient populations. This chapter also illustrates the variety of questions that are vital to ensuring effective use of medical interventions and how these issues might require trials with ever-increasing sample sizes that can be completed in a reasonable time period.



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2 Cases in Point: Learning from Experience INTRODUCTION Media accounts of medical research breakthroughs are full of examples of trial and study results that make headlines because of their potential to improve patient health or even save lives—but those headlines are sometimes misleading or limited in relevance to real-world care. This cycle has caused confusion and distrust among patients and consumers of health care. At the other end of the spectrum, researchers discount the value of some methodologies used to evaluate clinical effectiveness. In part these findings reflect the constantly evolving nature of scientific inquiry; but as illustrated in this chapter, these experiences offer lessons on the improvements needed in the design and interpretation of clinical effectiveness studies. By reviewing examples of high-profile studies and trials that evaluated the effectiveness of hormone replacement therapy, drug-eluting coronary stents, bariatric surgery, antipsychotic medications, and lung cancer screen- ing, this chapter illustrates the range of issues facing current effectiveness research. Examples of these issues include capturing important health out- comes throughout the lifecycle of an intervention; contending with the biologic complexity of disease and disease progression and rapid evolution of devices or surgical procedures or rapid uptake and application in broader patient populations. This chapter also illustrates the variety of questions that are vital to ensuring effective use of medical interventions and how these issues might require trials with ever-increasing sample sizes that can be completed in a reasonable time period. 

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 REDESIGNING THE CLINICAL EFFECTIVENESS RESEARCH PARADIGM The strengths and weaknesses of observational studies and random- ized trials are reviewed. Also reviewed are well-recognized limitations of observational studies due to the potential for confounding by a variety of factors as well as their limited capacity to assess short-term or acute risks. Although randomized controlled trials (RCTs) have the advantage of mini- mizing confounding, RCTs are often constrained by higher costs, shorter duration of follow-up, and limited applicability to populations of greatest clinical relevance. However, mixed experiences with different investigative approaches do not argue for total cessation of any one approach in favor of another. Rather, as the authors in this chapter suggest, the research com- munity needs to be more receptive to the use of alternative methodologies to generate insights into clinical effectiveness, and we need to determine which approach we use for a given question with full recognition of what is right for particular research circumstances. Collectively these experi- ences suggest the availability of a powerful array of methods, and when results are combined they produce more nuanced information needed to guide treatment decisions. Opportunities to strengthen these methods are discussed and, overall, greater attention is needed to define state-of-the-art methods so the quality of research is readily discernible regardless of study approach. In addition to methods, data and data system improvements are needed. Electronic health records and data registry approaches offer the opportunity to better systematically capture, track, and report outcomes. Moreover, there is the suggestion that a mix of research approaches, using the best advantages of particular designs, offers untapped promise and that researchers should be more open to adopting such approaches. Greater engagement by the healthcare system is imperative in the evaluation of effectiveness. JoAnn E. Manson from Harvard Medical School reviews the divergent results of observational studies and RCTs, evaluating the effect of meno- pausal hormone replacement therapy (HRT) on coronary heart disease (CHD). Despite this divergence, both have contributed critically important information on the therapies’ effectiveness and implications for healthcare decision making. Building on this experience, Manson discusses factors that might have contributed to the different findings. She suggests that because the short- and long-term effects of a clinical intervention may differ, both observational studies and clinical trial design must have benefits to offer researchers. Perhaps, she says, we should consider research findings in the context of all of the available evidence and design studies to complement and extend existing data. Large-scale studies involving networks of elec- tronic databases could facilitate evidence development. Due to the high cost and generally short duration of clinical trials, information about long-term risk may rely heavily on observational sources. The Food and Drug Administration’s (FDA’s) Ashley B. Boam recounts

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9 CASES IN POINT the differences in findings between initial pivotal clinical studies of drug- eluting coronary stents and subsequent studies using other methodology. She observes that further understanding of drug-eluting stents (DES) will likely come from a mix of randomized trials and observational registries, conducted both premarket and postmarket and involving a collaborative effort among regulators, industry, and academia. The next author, David R. Flum, a surgeon from the University of Washington, discusses the dichot- omy between “effectiveness” and “efficacy” and the applicability of case series in the context of bariatric surgical interventions. He concludes that population-based registries—appropriately funded and constructed with clinician engagement—offer a compromise of strengths and limitations and may be the most effective tool for evaluating emerging healthcare technology. Philip S. Wang from the National Institute of Mental Health (NIMH) discusses the recently completed, NIMH-sponsored comparative effective- ness trials of antipsychotic medications in patients with schizophrenia (the CATIE trial) as a model for a hybrid approach to study design that blends advantageous features of efficacy studies and large, simple trials. Wang reviews new data resources that may offer important opportunities to effectiveness research, practical clinical trials, adaptive designs, and cluster randomization when trials are not feasible, affordable, or in some cases, ethical. In the context of cancer research, Peter B. Bach from Memorial Sloan- Kettering Cancer Center discusses issues in the evaluation of screening tests, particularly the use of surrogate measures of benefit. He reviews the results of a computer simulation model to determine the value of lung cancer screening tests to illustrate some of the key challenges and the need for better approaches to ensure the consistent evaluation of the effectiveness of screening tests prior to widespread adoption. In particular, he suggests the use of coverage and payment as effective means to generate population- based longitudinal data on outcomes among screened groups. HORMONE REPLACEMENT THERAPY JoAnn E. Manson, M.D., Dr.P.H. Harvard Medical School Observational studies and randomized clinical trials of menopausal hormone therapy (HT) and coronary heart disease have produced widely divergent results. In aggregate, observational studies indicate that women who take estrogen after menopause are 35–50 percent less likely to develop CHD than women who do not take estrogen (Grodstein and Stampfer, 2002), whereas randomized trials suggest a neutral or even elevated risk

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90 REDESIGNING THE CLINICAL EFFECTIVENESS RESEARCH PARADIGM of coronary events with menopausal HT (Anderson et al., 2004; Hulley et al., 1998; Manson et al., 2003; Rossouw et al., 2002). The cardiovascular findings from the two HT trials (estrogen plus progestin and estrogen- alone) in the Women’s Health Initiative (WHI) are presented in Table 2-1. Understanding the basis for the discordant findings may provide impor- tant lessons for the design of future studies and may suggest strategies for improving the reliability and quality of clinical research. Detailed analyses from observational studies and randomized clinical trials have elucidated both methodological and biological explanations for the divergent findings, suggesting avenues for additional research to advance evidence develop- ment and improve clinical decision making (Grodstein et al., 2000, 2003; Manson and Bassuk, 2007b; Manson et al., 2006; Michels and Manson, 2003; Prentice et al., 2006). It is hoped that lessons learned from the dis- crepant results, which have provided insights into the strengths and weak- nesses of different sources of evidence, will serve as a springboard to the development of more reliable, efficient, and innovative designs for evaluat- ing clinical interventions. Methodological Factors That Contribute to the Divergent Findings The potential role of methodologic factors must be considered in under- standing the more favorable findings for HT in relation to CHD risk in observational studies than in clinical trials (Table 2-2). Well-recognized limitations of observational studies, including the potential for confound- ing by lifestyle practices, socioeconomic status, education, and access to medical care, as well as selection factors related to “indications for use,” can explain some—but not all—of the discrepancies (Grodstein et al., 2003; Manson et al., 2006; Michels and Manson, 2003; Prentice et al., 2006). TABLE 2-1 Hazard Ratios and 95 Percent Confidence Intervals for Cardiovascular Outcomes and Total Mortality in the Overall Study Population of Women Aged 50–79 in the Women’s Health Initiative (WHI) Trials of Menopausal Hormone Therapy Estrogen + Progestin Estrogen alone WHI Hormone Therapy Trials (N = 16,608) (N = 10,739) Coronary heart disease 1.24 (1.00–1.54) 0.95 (0.78–1.16) Stroke 1.31 (1.03–1.68) 1.33 (1.05–1.68) All-cause mortality 1.00 (0.83–1.19) 1.04 (0.88–1.22) SOURCES: Derived from Manson, J. E., J. Hsia, K. C. Johnson, et al. 2003. Estrogen plus pro- pro- gestin and the risk of coronary heart disease. New England Journal of Medicine 349:523-534; Rossouw, J. E., R. L. Prentice, J. E. Manson, et al. 2007. Postmenopausal hormone therapy and risk of cardiovascular disease by age and years since menopause. Journal of the American Medical Association 297:1465-1477.

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9 CASES IN POINT TABLE 2-2 Postmenopausal Hormone Therapy and CHD: Potential Explanations for Divergent Findings from Clinical Trials and Observational Studies Potential Explanations for the Divergent Findings • Methodological Differences — Confounding (“healthy user”) bias — Compliance bias — Incomplete capture of early clinical events • Biological Differences — Characteristics of study population (time since menopause, stage of atherosclerosis) — Hormone regimen (formulation and dose) SOURCE: Derived from Grodstein, F., T. B. Clarkson, and J. E. Manson. 2003. Understanding the divergent data on postmenopausal hormone therapy. New England Journal of Medicine 348:645-650. Confounding by healthful lifestyle practices and “healthy user bias” among women taking HT may have led to an overestimation of the CHD benefits, but most studies examining this issue suggest that careful adjust- ments for these factors (such as smoking, other CHD risk factors, body mass index, physical activity, and diet) attenuate—but do not eliminate— the inverse associations between HT and CHD risk (Grodstein et al., 2000, 2003; Manson et al., 2006). Moreover, the Nurses’ Health Study, a large- scale cohort relatively homogeneous for educational attainment, occupa- tion, and access to medical care, showed substantial reductions in CHD risk among HT users, compared to nonusers (Grodstein et al., 2000). Another methodologic factor that has received less attention is the limi- tation of most observational studies in assessing short-term or acute risks (due to infrequent updates of exposures), leading to incomplete capture of early clinical events after initiation of therapy and the predominance of follow-up time among compliant long-term users of HT (Grodstein et al., 2003; Manson and Bassuk, 2007b). In contrast to the greater weight- ing of long-term use in observational studies, clinical trial results tend to reflect shorter term use. Given that CHD risks related to HT are greatest soon after initiation of therapy (Hulley et al., 1998; Manson et al., 2003) and reductions in risk may emerge with longer term use (discussed below) (Michels and Manson, 2003; Prentice et al., 2006; Rossouw et al., 2002), these differences may contribute to the discrepancies observed. Indeed, comparative analyses of HT and CHD in the observational and clinical trial components of the Women’s Health Initiative, with stratification by dura- tion of treatment (comparing short-term versus long-term users), indicated greater convergence of study results when examining similar durations of

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92 REDESIGNING THE CLINICAL EFFECTIVENESS RESEARCH PARADIGM use (Prentice et al., 2006). For example, both the observational and clinical trial cohorts in the WHI suggested an increased risk of CHD during the first several years of HT use but a reduced risk with longer duration (>5 years) of use (Table 2-3). Moreover, observational studies that have utilized electronic health and pharmacy records, which provide frequent updating of exposure (HT use) information via prescription records and facilitate the capture of both short- and long-term health outcomes, have tended to show less pronounced reductions in CHD risk related to HT use (Heckbert et al., 2001; Lemaitre et al., 2006). In a study utilizing computerized pharmacy records and out- comes databases (Group Health Cooperative), the associations between HT and CHD risk were similar to those observed in the WHI for women of comparable age and health status (Heckbert et al., 2001). However, sub- stantial reductions in mortality among women with long-term use of HT have been observed even in studies using electronic pharmacy and health records (Ettinger et al., 1996). Thus, methodologic differences between observational studies and clin- ical trials may not fully elucidate the basis for the discrepancies observed. Although large-scale randomized trials, the gold standard of clinical research, have the advantage of minimizing confounding by lifestyle prac- tices, socioeconomic status, and other factors, they are often constrained by higher costs, shorter duration of follow-up, and, at times, limited applica- bility to populations of greatest clinical relevance. Furthermore, the findings of observational studies and clinical trials of HT are remarkably concordant TABLE 2-3 Hazard Ratios (HR) and 95 Percent Confidence Intervals (CIs) in the Women’s Health Initative (WHI), According to Duration of HT Use Comparison of Results from the WHI Clinical Trial (CT) and Observational Study (OS) for HT and CHD, According to Duration of Use Estrogen + Progestin Estrogen Alone HR (95% CI) HR (95% CI) Years since HT Initiation CT OS CT OS <2 1.68 1.12 1.07 1.20 (1.15–2.45) (0.46–2.74) (0.68–1.68) (0.49–2.94) 2–5 1.25 1.05 1.13 1.09 (0.87–1.79) (0.70–1.58) (0.70–1.58) (0.75–1.60) >5 0.66 0.83 0.80 0.73 (0.36–1.21) (0.67–1.01) (0.57–1.12) (0.61–0.84) SOURCE: Derived from Prentice, R., R. D. Langer, M. L. Stefanick, et al. 2006. Combined analysis of Women’s Health Initiative observational and clinical trial data on postmeno- pausal hormone treatment and cardiovascular disease. American Journal of Epidemiology 163(7):589-599.

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9 CASES IN POINT Coronary hear t disease Observational studies WHI E + P Stroke Observational studies WHI E + P Pulmonar y embolism Observational studies WHI E + P Hip fractures Observational studies WHI E + P Breast cancer Observational studies WHI E + P Colorectal cancer Observational studies WHI E + P 0 0.5 1 1.5 2.0 2.5 3.0 3.5 4.0 Relative Risk FIGURE 2-1 Relative risks and 95 percent confidence intervals for observational and clinical trial findings on hormone therapyeps Figure 2-1. (estrogen + progestin). SOURCE: Michels, K. B., and J. E. Manson. 2003. Postmenopausal hormone therapy: A reversal of fortune. Circulation 107:1830-1833. Reprinted with permis- sion from Michels and Manson, Circulation, 2003. for non-CHD health outcomes, including stroke, venous thromboembolism, breast cancer, colorectal cancer, and fracture (Figure 2-1)—results that should also be affected by confounding and selection biases (Grodstein et al., 2003; Manson et al., 2006; Michels and Manson, 2003). An emerging body of evidence supports the hypothesis that age or time since menopause critically influences the relationship between HT and CHD outcomes (Estrogen and progestogen use in peri- and postmenopausal women: March 2007 position statement of the North American Menopause Society, 2007; Grodstein et al., 2003; Manson et al., 2006). Women who participate in observational studies tend to be younger and closer to onset of menopause at the time of HT initiation than women in randomized trials (the latter are, on average, more than a decade past menopause onset at randomization). Thus, women in HT clinical trials tend to have later stages of atherosclerosis than their counterparts in observational studies and a possibly greater vulnerability to the adverse vascular effects of HT (Estrogen and progestogen use in peri- and postmenopausal women: March 2007 position statement of the North American Menopause Society, 2007; Manson et al., 2006). In contrast, if estrogen slows early stages of athero- sclerosis, as suggested by basic research, animal studies, and imaging find- ings, recently menopausal women with healthy vascular endothelium may be more likely to have a favorable coronary outcome than women more

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9 REDESIGNING THE CLINICAL EFFECTIVENESS RESEARCH PARADIGM distant from menopause (Manson et al., 2007; Mendelsohn and Karas, 2005; Mikkola and Clarkson, 2002). Moreover, absolute rates of adverse events and risks attributable to HT are lower in younger than older women, suggesting that the risk:benefit ratio may vary substantially by age and proximity to menopause onset (Estrogen and progestogen use in peri- and postmenopausal women: March 2007 position statement of the North American Menopause Society, 2007; Manson and Bassuk, 2007b). It is important to emphasize that the implication of the “timing hypothesis” is not that recently menopausal women be given HT for CHD preven- tion but rather that clinicians can be reassured about cardiac risks when considering short-term use of HT for vasomotor symptom management in such women. The theory that the influence of estrogen on atherosclerosis and coronary events may vary according to the underlying health of the vasculature and the evidence that a woman’s age and time since menopause onset may modulate CHD outcomes with HT, as well as implications for future research, are discussed below. Biological Factors That May Contribute to the Divergent Findings As noted above, randomized trials testing the effect of HT on clinical coronary outcomes have not confirmed the cardioprotective effect sug- gested by most observational studies. In the Heart and Estrogen/progestin Replacement Study (HERS), the 4-year incidence of major coronary events among women with a mean age of 67 years and with preexisting CHD was similar in the HT (oral conjugated equine estrogens [CEE] and medroxy- progesterone acetate [MPA]) and the placebo groups (Hulley et al., 1998). The HT group had a 50 percent increase in risk of CHD events during the first year of the trial, although this elevation was offset by a decreased risk in later years (Grady et al., 2002; Hulley et al., 1998). The Women’s Health Initiative examined the effects of oral CEE with or without MPA in healthy postmenopausal women aged 50–79 (mean age 63) (Anderson et al., 2004; Rossouw et al., 2002); participants had either an intact uterus (N = 16,608) or prior hysterectomy (N = 10,739), respectively. Women assigned to CEE + MPA for an average of 5.6 years were more likely to experience a CHD event than those assigned to placebo (relative risk [RR] = 1.24; 95 percent confidence interval [CI]: 1.00, 1.54), with the highest risk during the first year (Manson et al., 2003). Women assigned to CEE alone for an average of 6.8 years also experienced no overall reduction in CHD risk (RR = 0.95; 95 percent CI: 0.78, 1.16) (Prentice et al., 2006). Both WHI trials were stopped early—the CEE + MPA trial because of an increased risk of breast cancer and an unfavorable benefit–risk balance (Rossouw et al., 2002) and the CEE-alone trial because of an increased stroke risk that was not offset by a reduced CHD risk (Anderson et al., 2004). Although most randomized

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9 CASES IN POINT clinical trials have tested the commonly used HT formulations of CEE + MPA or CEE alone, clinical trials using estradiol and other formulations of estrogen and/or progestin have also failed to demonstrate cardioprotection (Grodstein et al., 2003; Manson et al., 2006; Michels and Manson, 2003). A key difference between participants in observational studies and those in clinical trials of HT is the timing of initiation of treatment in relation to menopause onset, which occurs on average at age 51 in the United States. Hormone users in observational studies typically start therapy in early menopause, whereas trial participants are often randomized to hormones more than a decade after cessation of menses. For example, in the Nurses’ Health Study cohort, about 80 percent of women who used HT began treatment within 2–3 years of menopause onset (Grodstein et al., 2003; Manson and Bassuk, 2007b). In contrast, WHI participants, with a mean baseline age of 63, were an average of at least 12 years past menopause at the time of trial enrollment and likely had more extensive atherosclerosis than newly menopausal women. In HERS, the mean age was 67 at baseline, and all participants had been previously diagnosed with CHD. It has been hypothesized that estrogen has diverse and opposing actions, slowing the earlier stages of atherosclerosis through favorable effects on the lipid pro- file and endothelial function, but triggering acute coronary events through prothrombotic and inflammatory mechanisms and plaque destabilization when advanced lesions are present (Estrogen and progestogen use in peri- and postmenopausal women: March 2007 position statement of the North American Menopause Society, 2007; Grodstein et al., 2003; Manson et al., 2006; Mendelsohn and Karas, 2005) (Figure 2-2). This hypothesis is supported by several lines of evidence from basic and clinical studies. First, trials in humans show complex effects of exogenous estrogen on cardiovascular biomarkers (Estrogen and progesto- gen use in peri- and postmenopausal women: March 2007 position state- ment of the North American Menopause Society, 2007; Manson et al., 2006; Mendelsohn and Karas, 2005). Oral estrogen lowers low-density lipoprotein (LDL) cholesterol, lipoprotein(a), glucose, insulin, and homo- cysteine levels; inhibits oxidation of LDL cholesterol; increases high-density lipoprotein cholesterol; reverses postmenopausal increases in fibrinogen and plasminogen-activator inhibitor type 1; and improves endothelial function—all effects expected to lower coronary risk. However, oral estro- gen also increases triglycerides, coagulation factors (factor VII, prothrombin fragments 1 and 2, and fibrinopeptide A), C-reactive protein, and matrix metalloproteinases—effects expected to raise coronary risk. Additionally, certain progestogens may offset some of estrogen’s benefits. Data from controlled experiments in nonhuman primates also support the theory that the coronary effects of HT depend on the initial health of the vasculature. Conjugated estrogen (with or without a progestin) did not

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Premenopausal Years Postmenopausal Years 9 Years Since Menopause Onset 5 10 15 20 Observational Studies Clinical Trials (1° and 2° Prevention) Progression of Cardiovascular Disease Fatty streaks Fatt y plaques Atherosclerotic plaques Plaques grow and may rupture Favorable Lipid and Prothrombotic and Endothelial Effects of Proinflammatory Effects of Estrogen Predominate Estrogen Predominate (plaque rupture, thrombo- occlusion) Adverse Influence Favorable Influence of Initiating of Initiating Exogenous Estrogens Exogenous Estrogens FIGURE 2-2 Schematic illustration of the interrelationships of timing of initiation of hormone therapy, vascular health, and clinical CHD outcomes. Figure 2-2.eps SOURCE: Manson, J. E., S. S. Bassuk, S. M. Harman, et al. 2006. Postmenopausal hormone therapy: New questions and the case bitmap image with vector elements for new clinical trials. Menopause 13(1):139-147. landscape

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9 CASES IN POINT affect the extent of coronary artery plaque in cynomolgus monkeys started on this treatment at 2 years (~6 human years) after oophorectomy and well after the establishment of atherosclerosis, but such therapy reduced plaque by 70 percent when initiated immediately after oophorectomy during the early stages of atherosclerosis (Mikkola and Clarkson, 2002) (Figure 2-3). Similarly, imaging trials in women with significant coronary lesions at baseline have found estrogen to be ineffective in slowing the rate of arte- rial narrowing (Angerer et al., 2001; Herrington et al., 2000; Hodis et al., 2003; Waters et al., 2002). However, in an imaging trial that did not require participants to have significant vascular disease at entry, estrogen impeded progression of carotid atherosclerosis (Hodis et al., 2001). When the WHI trials were initiated in the early 1990s, it was not well recognized that age or vascular health might be an important deter- minant of the effect of HT on coronary or other outcomes; thus, focused subgroup analyses were not emphasized at the outset, nor were the trials powered to detect potential interactions. However, given the striking dis- crepancies between findings from earlier observational studies and more recent randomized trials (including data from the large trials with hard clinical endpoints, smaller imaging studies, and experimental studies in animals), WHI investigators pursued more detailed analyses of the data to examine whether the timing hypothesis might account for the seemingly contradictory evidence on coronary effects of HT. OVARIECTOMY Plaque area reduction Healthy Diet CEE + Atherogenic Diet 70 % Atherogenic CEE + Healthy 0% Healthy Diet Diet Diet vs. monkeys TIME getting no CEE Premenopause Perimenopause Postmenopause FIGURE 2-3 Role of timing of conjugated equine estrogen (CEE) initiation in rela- tionship to ovariectomy in nonhuman primates. s Figure 2-3.ep NOTE: Modified and reprinted with permission from the European Society of Cardiology, Copyright © 2002. SOURCE: Mikkola, T. S., and T. B. Clarkson. 2002. Estrogen replacement therapy, atherosclerosis, and vascular function. Cardiovasc Res 53:605-619.

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