Clinical Management of Pediatric Dyslipidemia
The modern practice of medicine is directed, whenever possible, by published evidence to ensure efficacy while avoiding the potential for harm. Although different types of medical evidence exist, randomized controlled trials (RCTs) are considered the gold standard. In the absence of published evidence, practitioners in general rely on consensus opinions from experts in the field. Evidence-based guidelines and consensus statements often are issued by professional societies and organizations.
The NHLBI selected a highly qualified panel of noted experts who carefully and methodically examined the available evidence pertinent to cardiovascular health and risk reduction in children and adolescents. Although RCTs were included in the Expert Panel reviews, notably absent were the results of long-term RCTs of safety and efficacy. The latter are problematic in children because such trials take a long time to complete, given the extensive interval between initial identification of CVD risk factors and disease expression. This is particularly true when measuring common adult CVD study "hard endpoints" such as myocardial infarction and death. Furthermore, given the extended period of time to reach these endpoints, lifestyles and therapeutic options would likely change. Finally, given the significant benefit of statins in reducing CVD-related mortality in adults, withholding effective treatment in moderate- to high-risk children, such as those with FH, would be concerning to many healthcare providers.
Given these constraints, the Expert Panel extensively reviewed epidemiological, observational, and case-control studies in formulating its recommendations for youth. Only time and continued surveillance of the pediatric population at risk will tell whether these recommendations are flawed. Given the serious consequences of unrecognized and/or inadequately managed CVD risk factors as well as the known safety profile of statins in children, such recommendations seem warranted.
Concerns and Controversy
The modern practice of medicine is directed, whenever possible, by published evidence to ensure efficacy while avoiding the potential for harm. Although different types of medical evidence exist, randomized controlled trials (RCTs) are considered the gold standard. In the absence of published evidence, practitioners in general rely on consensus opinions from experts in the field. Evidence-based guidelines and consensus statements often are issued by professional societies and organizations.
The NHLBI selected a highly qualified panel of noted experts who carefully and methodically examined the available evidence pertinent to cardiovascular health and risk reduction in children and adolescents. Although RCTs were included in the Expert Panel reviews, notably absent were the results of long-term RCTs of safety and efficacy. The latter are problematic in children because such trials take a long time to complete, given the extensive interval between initial identification of CVD risk factors and disease expression. This is particularly true when measuring common adult CVD study "hard endpoints" such as myocardial infarction and death. Furthermore, given the extended period of time to reach these endpoints, lifestyles and therapeutic options would likely change. Finally, given the significant benefit of statins in reducing CVD-related mortality in adults, withholding effective treatment in moderate- to high-risk children, such as those with FH, would be concerning to many healthcare providers.
Given these constraints, the Expert Panel extensively reviewed epidemiological, observational, and case-control studies in formulating its recommendations for youth. Only time and continued surveillance of the pediatric population at risk will tell whether these recommendations are flawed. Given the serious consequences of unrecognized and/or inadequately managed CVD risk factors as well as the known safety profile of statins in children, such recommendations seem warranted.