Statins for children with familial hypercholesterolemia

Vuorio, Alpo; Kuoppala, Jaana; Kovanen, Petri T.; Humphries, Steve E.; Tonstad, Serena; Wiegman, Albert; Drogari, Euridiki; Ramaswami, Uma

doi:10.1002/14651858.cd006401.pub4

Cited by 131 publications

(113 citation statements)

References 107 publications

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“…Data from the AdDIT trial have also provided support for the wider use of statins in young people (>10 years of age) with T1DM in whom high lipid levels are detected. The studies provided reassuring safety data for this patient population, which are similar to those previously reported in children and adolescents with familial hypercholesterolemia 75 . Furthermore, these high risk young individuals might be potential candidates for new reno-protective drugs, such as Sodium-glucose Cotransporter-2 inhibitors, which have shown promising results in terms of cardio-renal outcomes in adults with diabetes 76 .…”

Section: Current Guidelinessupporting

confidence: 82%

A new strategy for vascular complications in young people with type 1 diabetes mellitus

et al. 2019

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on behalf of the Adolescent type 1 Diabetes cardio-renal Intervention Trial (AdDIT) study group Abstract Diabetes vascular complications, including cardiovascular disease, diabetic nephropathy and retinopathy, have a negative effect on the long-term prognosis of young people with type 1 diabetes mellitus. Poor glycaemic control and consequent increased HbA1c levels are major risk factors for the development of vascular complications. HbA1c levels are the main focusof current management strategies, however, recommended target is rarely achieved in adolescents. Thus, a clear need exists for improved biomarkers to identify high risk young people at an early stage and to develop new intervention strategies. Evidence is accumulating that early increases in urinary albumin excretion could be predictive of adolescents with type 1 diabetes who are at an increased risk of developing vascular complications, independently of HbA1c levels. These findings present an opportunity to move towards the personalized care of adolescents with type 1 diabetes, which takes into consideration changes in albumin excretion and other risk factors, in addition to HbA1c

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Section: Current Guidelinessupporting

confidence: 82%

A new strategy for vascular complications in young people with type 1 diabetes mellitus

et al. 2019

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“…The magnitude of this reduction is similar to the reductions achieved in rigorous short and medium term controlled trials, demonstrating that clinical trial success in reducing LDL-C in children and adolescents is possible in real world clinical environments. A Cochrane review of clinical trials on the use of statins in children with FH identified 5 trials involving 566 children; the meta-analysis results showed a mean decrease at the end of follow-up (median 24 weeks) of 32% [95% 29–35] (10). Reporting from a clinical setting, Carreau et al completed an audit of 185 children and teenagers with dyslipidemia followed from three months to seven years between 2002 and 2009 (26).…”

Section: Discussionmentioning

confidence: 99%

“…Short and medium term randomized controlled trials of statins in children with FH have demonstrated decreases in LDL-C by 20–40% (8–10). Data are lacking about the efficacy of statin therapy in achieving LDL-C goals in children and adolescents outside of a clinical trial environment.…”

mentioning

confidence: 99%

Correlates of Achieving Statin Therapy Goals in Children and Adolescents with Dyslipidemia

Mendelson

Regh

Chan

et al. 2016

The Journal of Pediatrics

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Objective To determine the real world effectiveness of statins and impact of baseline factors on low-density lipoprotein cholesterol (LDL-C) reduction among children and adolescents. Study design We analyzed data prospectively collected from a quality improvement initiative in the Boston Children’s Hospital Preventive Cardiology Program. We included patients ≤ 21 years of age initiated on statins between September 2010 and March 2014. The primary outcome was first achieving goal LDL-C, defined as <130 mg/dL, or <100 mg/dL with high-level risk factors (e.g. diabetes, etc.). Cox proportional hazards models assessed the impact of baseline clinical and lifestyle factors. Results Among the 1521 pediatric patients evaluated in 3813 clinical encounters over 3.5 years, 97 patients (6.3%) were started on statin therapy and had follow-up data (median age 14 [IQR 7] years), 54% were female, 24% obese, 62% with at least one lifestyle risk factor. The median baseline LDL-C was 215 (IQR 78) mg/dL and median follow-up after starting statin was 1.0 (IQR 1.3) year. The cumulative probability of achieving LDL-C goal within 1 year was 60% (95% CI 47, 69). Male sex (HR 0.5 [95% CI 0.3, 0.8]) and higher baseline LDL-C (HR 0.92 [95% CI 0.87, 0.98] per 10 mg/dL) were associated with not achieving LDL-C goals, but not age, BMI percentile, lifestyle factors or family history. Conclusions The majority of pediatric patients started on statins reached LDL-C treatment goals within 1 year. Males and those with higher baseline LDL-C were less likely to be successful and may require increased support.

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“…34 A 2017 Cochrane review analysed nine randomised controlled trials comparing the efficacy and safety of statins versus placebo in 1177 children with FH aged 6 to 18 years; the authors concluded that statins seem to be safe in the short term, but long-term safety remains unknown. 35 Patients are initially treated with the lowest doses, which can be increased as necessary. Some patients are prescribed bile acid sequestrants (eg, colestyramine) as early as age 1 year, and ezetimibe at age ≥10 years (…”

Section: Pharmacotherapymentioning

confidence: 99%

Guidance on the management of familial hypercholesterolaemia in Hong Kong: an expert panel consensus viewpoint

Tomlinson

Chan

Chan³

et al. 2018

Hong Kong Med J

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In 2016, meetings of groups of physicians and paediatricians with a special interest in lipid disorders and familial hypercholesterolaemia were held to discuss several domains of management of familial hypercholesterolaemia in adults and children in Hong Kong. After reviewing the evidence and guidelines for the diagnosis, screening, and management of familial hypercholesterolaemia, consensus was reached on the following aspects: clinical features, diagnostic criteria, screening in adults, screening in children, management in relation to target plasma low-density lipoprotein cholesterol levels, detection of atherosclerosis, lifestyle and behaviour modification, and pharmacotherapy.

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Statins for children with familial hypercholesterolemia

Cited by 131 publications

References 107 publications

A new strategy for vascular complications in young people with type 1 diabetes mellitus

A new strategy for vascular complications in young people with type 1 diabetes mellitus

Correlates of Achieving Statin Therapy Goals in Children and Adolescents with Dyslipidemia

Guidance on the management of familial hypercholesterolaemia in Hong Kong: an expert panel consensus viewpoint

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