Screening for Lipid Disorders in Children and Adolescents

Abstract

ImportanceLipid screening in childhood and adolescence can lead to early dyslipidemia diagnosis. The long-term benefits of lipid screening and subsequent treatment in this population are uncertain.ObjectiveTo review benefits and harms of screening and treatment of pediatric dyslipidemia due to familial hypercholesterolemia (FH) and multifactorial dyslipidemia.Data SourcesMEDLINE and the Cochrane Central Register of Controlled Trials through May 16, 2022; literature surveillance through March 24, 2023.Study SelectionEnglish-language randomized clinical trials (RCTs) of lipid screening; recent, large US cohort studies reporting diagnostic yield or screen positivity; and RCTs of lipid-lowering interventions.Data Extraction and SynthesisSingle extraction, verified by a second reviewer. Quantitative synthesis using random-effects meta-analysis.Main Outcomes and MeasuresHealth outcomes, diagnostic yield, intermediate outcomes, behavioral outcomes, and harms.ResultsForty-three studies were included (n = 491 516). No RCTs directly addressed screening effectiveness and harms. Three US studies (n = 395 465) reported prevalence of phenotypically defined FH of 0.2% to 0.4% (1:250 to 1:500). Five studies (n = 142 257) reported multifactorial dyslipidemia prevalence; the prevalence of elevated total cholesterol level (≥200 mg/dL) was 7.1% to 9.4% and of any lipid abnormality was 19.2%. Ten RCTs in children and adolescents with FH (n = 1230) demonstrated that statins were associated with an 81- to 82-mg/dL greater mean reduction in levels of total cholesterol and LDL-C compared with placebo at up to 2 years. Nonstatin-drug trials showed statistically significant lowering of lipid levels in FH populations, but few studies were available for any single drug. Observational studies suggest that statin treatment for FH starting in childhood or adolescence reduces long-term cardiovascular disease risk. Two multifactorial dyslipidemia behavioral counseling trials (n = 934) demonstrated 3- to 6-mg/dL greater reductions in total cholesterol levels compared with the control group, but findings did not persist at longest follow-up. Harms reported in the short-term drug trials were similar in the intervention and control groups.Conclusions and RelevanceNo direct evidence on the benefits or harms of pediatric lipid screening was identified. While multifactorial dyslipidemia is common, no evidence was found that treatment is effective for this condition. In contrast, FH is relatively rare; evidence shows that statins reduce lipid levels in children with FH, and observational studies suggest that such treatment has long-term benefit for this condition.

Pediatric Lipid Screening Prevalence Using Nationwide Electronic Medical Records

“…31,32 The USPSTF evidence reviews from 2016 and 2023 summarized randomized clinical trial data and found no direct evidence of either effectiveness or harms of universal lipid screening in youths. 17,18 In contrast to AAP recommendations endorsing universal lipid screening, the USPSTF conclusion likely added confusion for clinicians regarding the necessity of cholesterol screening, which may negatively impact screening rates.…”

“…Although universal lipid screening recommendations have been endorsed by the AAP since 2011, our findings may indicate that (1) health care professionals may not have a clear understanding of the childhood screening guidelines or (2) there may be barriers to changing screening and prevention practices . The USPSTF evidence reviews from 2016 and 2023 summarized randomized clinical trial data and found no direct evidence of either effectiveness or harms of universal lipid screening in youths . In contrast to AAP recommendations endorsing universal lipid screening, the USPSTF conclusion likely added confusion for clinicians regarding the necessity of cholesterol screening, which may negatively impact screening rates.…”

“…8 In 2016 and 2023, the US Preventive Services Task Force (USPSTF) conducted evidence reviews to examine lipid screening in children and adolescents. 17,18 They were unable to identify any studies that demonstrated that screening for dyslipidemia in asymptomatic children or adolescents delayed or reduced the incidence of CVD or mortality, nor were they able to identify any studies that demonstrated harms of screening. They concluded that there is still insufficient evidence to assess the balance of benefits or harms of lipid screening among children.…”

“…They concluded that there is still insufficient evidence to assess the balance of benefits or harms of lipid screening among children. 17,18 Despite these screening recommendations, studies have shown low pediatric lipid screening rates, including among youths with higher-risk conditions such as obesity. 7,10,19 However, prior studies examining screening prevalence are limited in number and recency of data.…”

“…They were unable to identify any studies that demonstrated that screening for dyslipidemia in asymptomatic children or adolescents delayed or reduced the incidence of CVD or mortality, nor were they able to identify any studies that demonstrated harms of screening. They concluded that there is still insufficient evidence to assess the balance of benefits or harms of lipid screening among children …”