Pediatric experience with mipomersen as adjunctive therapy for homozygous familial hypercholesterolemia.

Background: Homozygous familial hypercholesterolemia (HoFH) is a rare, inherited condition resulting in severely elevated low-density lipoprotein cholesterol levels (LDL-C) leading to premature cardiovascular disease and, often, death. Mipomersen is an antisense oligonucleotide that inhibits apolipoprotein B (apo B) synthesis, lowering LDL-C levels. Mipomersen has demonstrated efficacy in adult HoFH patients, possibly providing a therapeutic option for pediatric patients.

Population pharmacokinetics of rosuvastatin in pediatric patients with heterozygous familial hypercholesterolemia.

Purpose: Data from two clinical studies (hyperCholesterolaemia in cHildren and Adolescents taking Rosuvastatin OpeN label [CHARON; NCT01078675] and Study 4522IL/0086) were used to describe rosuvastatin pharmacokinetics in patients with heterozygous familial hypercholesterolemia aged ≥6 to <18 years.

Methods: Rosuvastatin concentration-time data were analyzed via non-linear mixed-effects modeling (NONMEM), with clearance (CL/F) as the pre-defined key pharmacokinetic parameter of interest. In addition, descriptive comparisons between pediatric patients and adults (healthy and dyslipidemic) were performed.

Gonadal steroids, gonadotropins and DHEAS in young adults with familial hypercholesterolemia who had initiated statin therapy in childhood.

Objective: Statins are currently the preferred pharmacological therapy in children with familial hypercholesterolemia (FH) with the aim to prevent premature cardiovascular disease (CVD). However, concerns have been raised that lowering cholesterol levels with statins could interfere with hormone production. In this study hormone concentrations were assessed in young adult FH subjects before and 10 years after the initiation of statins, and compared with their unaffected siblings.

Efficacy and Safety of Pitavastatin in Children and Adolescents at High Future Cardiovascular Risk.

Objectives: To assess the safety and efficacy of pitavastatin in children and adolescents with hyperlipidemia.

Study Design: A total of 106 children and adolescents with hyperlipidemia, ages 6 to 17 years, were enrolled in a 12-week randomized, double-blind, placebo-controlled study and randomly assigned to pitavastatin 1 mg, 2 mg, 4 mg, or placebo. During a 52-week extension period, subjects were up-titrated from 1 mg pitavastatin to a maximum dose of 4 mg in an effort to achieve an optimum low-density lipoprotein cholesterol (LDL-C) treatment target of <110 mg/dL (2.

Early initiation of statin treatment in children with familial hypercholesterolaemia.

Purpose Of Review: This article provides recent insights on the early onset of atherosclerosis in heterozygous familial hypercholesterolemia and reports on novel treatment options as well as on the consequences of long-term statin use in childhood.

Recent Findings: Children with familial hypercholesterolemia have greater mean carotid intima-media thickness (cIMT) than their unaffected siblings even before the age of 8 years, which is several years earlier than previously reported. In those children, 2 years of rosuvastatin treatment resulted in slowing of the cIMT progression.

Long-term statin treatment in children with familial hypercholesterolemia: more insight into tolerability and adherence.

Background: Statins are currently the preferred pharmacological therapy in individuals with familial hypercholesterolemia (FH) with the aim to prevent premature atherosclerosis. In adults, these agents have been proven to be safe and well tolerated; however, non-adherence is a significant clinical issue.

Objectives: In this study, we evaluated tolerability and adherence to statin therapy in young adult FH patients 10 years after this was initiated in their childhood.

Management of hypercholesterolemia in children.

Cardiovascular disease (CVD) remains the leading cause of death and morbidity in our society. One of the major risk factors for CVD is hypercholesterolemia. Hypercholesterolemia in children can be caused by a hereditary disorder or can be secondary to other diseases or drugs.

Statin therapy and secretory phospholipase A₂ in children with heterozygous familial hypercholesterolemia.

Objective: Secretory phospholipase A2 (sPLA2) enzymes are thought to contribute to atherosclerosis. In this study we assessed levels of sPLA2 mass and activity, and their relationship to baseline characteristics of children with familial hypercholesterolemia (FH). Furthermore, we evaluated the effect of two years of pravastatin therapy on sPLA2 levels.

Inheritance pattern of familial hypercholesterolemia and markers of cardiovascular risk.

Studies in children and adults have resulted in conflicting evidence in the quest for the answer to the hypothesis that offspring from hypercholesterolemic mothers might have an increased cardiovascular risk. Previous studies might have suffered from limitations such as cohort size and clinical sampling bias. We therefore explored this hypothesis in large cohorts of both subjects with familial hypercholesterolemia (FH) and unaffected siblings in a wide age range.

Drug therapy of hypercholesterolaemia in children and adolescents.

Cardiovascular disease (CVD) remains the leading cause of death and morbidity in the world. The origins of atherosclerosis and subsequent CVD begin in childhood. In order to prevent CVD, children and adolescents at high risk for premature atherosclerosis should be identified and treated as early as possible.

Clinical practice. Protein-losing enteropathy in children.

Protein-losing enteropathy (PLE) is a rare complication of a variety of intestinal disorders characterized by an excessive loss of proteins into the gastrointestinal tract due to impaired integrity of the mucosa. The clinical presentation of patients with PLE is highly variable, depending upon the underlying cause, but mainly consists of edema due to hypoproteinemia. While considering PLE, other causes of hypoproteinemia such as malnutrition, impaired synthesis, or protein loss through other organs like the kidney, liver, or skin, have to be excluded.