Don P. Wilson

Background and Aims: In patients with lysosomal acid lipase deficiency (LAL‐D), elevations in alanine and aspartate aminotransferases (ALT, AST) are associated with liver damage. The objective of this analysis was to evaluate aminotransferase levels in patients treated with sebelipase alfa enzyme replacement therapy and untreated patients. Methods: Patients in this observational study were from the International LAL‐D Registry and included untreated and treated patients who started sebelipase alfa at age ≥ 6 months. Patients in the longitudinal analysis must have had baseline and longitudinal measures of ALT or AST. A subanalysis was performed on data from patients with 3 consecutive annual follow‐up measures. Results: Of 186 patients included in this analysis, 125 were treated with sebelipase alfa. Median age at diagnosis was 9.1 y for treated patients and 20.6 y for untreated patients. Baseline ALT levels were above the upper limit of normal in 93% of children aged < 18 years and 68% of adults aged ≥ 18 years. Among 53 treated patients who had ALT results reported at 3 consecutive annual measurements, 22 (42%) had ALT levels within normal limits after 1 year of sebelipase alfa treatment. Results were similar for AST. A repeated measures regression model of data from treated patients showed significant reductions from baseline in both ALT and AST levels across 3 years of the study. Untreated patients had no appreciable changes in aminotransferase elevations over time. Conclusions: Aminotransferase levels were elevated in most patients with LAL‐D at baseline. There were sustained improvements with sebelipase alfa treatment. Trial Registration: International LAL‐D Registry: NCT01633489, EUPAS13276

The clustering of cardiometabolic risk factors, commonly referred to as metabolic syndrome (MetS), has become increasingly prevalent among youth <18 years of age. Although its definition in this population is often debated, as in adults MetS is typically characterized by central obesity, high blood pressure, dyslipidemia (elevated triglycerides and low high density lipoprotein cholesterol), and glucose dysregulation, with insulin resistance as the common link. MetS frequently originates in childhood, tracks into adulthood, and is associated with comorbidities such as fatty liver disease and polycystic ovarian syndrome. Furthermore, it significantly increases the risk of developing type 2 diabetes and cardiovascular disease later in life. In this article, we discuss MetS, its components, associated comorbidities, evaluation, and management strategies in the era of recent medications now approved for use in pediatrics. While short-term use of weight loss medications, such as glucagon-like peptide-1 receptor agonists, has been shown to be effective, the safety and long-term benefits have not been determined.

Purpose of Review We review treatment criteria in the pediatric population, provide practical advice on how and when to prescribe statins, and share tips to improve compliance. Recent Findings Although long-term outcome studies of cardiovascular-related events, such as myocardial infarction (MI) and stroke, are lacking in this population, statin therapy initiated during adolescence has been shown to be safe and effective for up to 20 years of continuous use. Summary HMG-CoA reductase inhibitors (statins) are the most effective class of drugs for lowering low-density lipoprotein cholesterol (LDL-C) in children and adolescents.

Purposeof Review A heart-healthy lifestyle adopted during childhood and sustained throughout life can reduce cardiovascular disease risk in youth with dyslipidemia. In this review, we discuss nutrition recommendations for youth (< 18 years-of-age) with dyslipidemia, compare recommendations for youth versus those for adults, review published data regarding nutrition management in the pediatric population, and discuss strategies for successful implementation in a clinical setting. Recent Findings Recent publications highlight the characteristics of genetic and acquired hypertriglyceridemia disorders, dietary adjuncts used for lipid-lowering, and the effectiveness of a multi-disciplinary team approach. Summary Nutrition interventions remain a cornerstone of lipid management and cardiovascular disease risk reduction in youth with dyslipidemia. Nutrition counseling should include age and developmentally appropriate education while also addressing barriers to implementing a heart-healthy lifestyle. A registered dietitian nutritionist plays an important role within a multidisciplinary clinic setting by providing dietary recommendations to address the needs of youth with dyslipidemia.