Article Archive

Familial hypercholesterolaemia (FH)

Why is cholesterol important? Cholesterol, with other fats such as triglycerides, plays a vital role in the structure and function of cells. However, too much cholesterol in the blood (hypercholesterolaemia) is a risk factor for early heart disease, heart attack and stroke. Cholesterol is transported…

FH issues in low to middle income regions

Despite the availability of statins, treatment of familial hypercholesterolaemia (FH) is a significant issue with a high unmet need in a middle-income country, according to Dr Dirk Blom of Cape Town, South Africa. As a result, new novel therapies are urgently needed.

Bococizumab

March 30, 2014 1183 – Prevention: Familial Hypercholesterolemia, Novel Therapies and CV Risk Efficacy and Safety of Bococizumab (RN316/PF-04950615), a Monoclonal Antibody against Proprotein Convertase Subtilisin/Kexin Type 9 in Statin-Treated Hypercholesterolemic Subjects: Results from a Randomized, Placebo-Controlled, Dose-Ranging Study (NCT: 01592240) Session 1183-129, 9:45 -…

Does PCSK9 inhibition on top of statin therapy offer the possibility of further reducing plaque burden?

Stephen J Nicholls MBBS PhD South Australian Health and Medical Research Institute, University of Adelaide, Australia Technical advances in arterial wall imaging have enabled the visualization of the full burden of atherosclerotic plaque. When applied in serial imaging studies of anatomically matched arterial segments, it…

The FH enigma: are there other FH-causing genes?

A genetic diagnosis is established in about 80% of patients with FH. The question is what is the genetic basis for FH in the remaining cases. A recent study1 used whole exome sequencing of FH patients negative for LDLR, APOB or PCSK9 mutations, in an…

PCSK9 and plaque: trials

Recent trials have shown that the PCSK9 monoclonal antibodies lower LDL cholesterol by more than 50% on top of statin therapy. There is also evidence that serum PCSK9 is associated with carotid intima-media thickness, a surrogate for atherosclerosis. Lee CJ, Lee YH, Park SW et…

Professor Gerald Watts discusses the 10 countries project in FH

The International Atherosclerosis Society has begun a study in Asia and the Pacific Rim to provide the first comprehensive investigation of the worlds commonest genetic disorder, familial hypercholesterolaemia (FH) in the region. FH results in very high levels of low density lipoprotein cholesterol (LDL-C) and…

Rising to the Challenge: Implementing Models of Care in FH

Winthrop Professor Gerald F. Watts DSc PhD MD FRACP FRCP, Lipid Disorders Clinic, Cardiovascular Medicine, Royal Perth Hospital School of Medicine and Pharmacology, University of Western Australia. GPO Box X2213, Perth, WA 6847, Australia; E-mail: [email protected] While several guidelines, mostly based on expert opinion, have…

PCSK9: Opportunities for personalised medicine?

Personalised medicine shifts the focus from a ‘one-size-fits-all’ to patient-centred alternatives. This undoubtedly benefits both patients and healthcare budgets. Such an approach has already been integrated into other disciplines, such as oncology. Is such a strategy also relevant in the management of familial hypercholesterolaemia (FH)?…

What are the current unmet needs?

Despite statins, there are significantly unmet clinical needs in cholesterol lowering treatment. Statin do not effectively treat more than 50% of people with Familial Hypercholesterolaemia (FH) and other patients have intolerable side effects, says Professor Erik Stroes.

Statins and plaque burden

The clinical benefit of LDL cholesterol lowering with statins has been conclusively demonstrated with a wealth of experience from primary and secondary prevention clinical trials.1,2 In addition, imaging studies have shown that statins have a favourable effect on atherosclerosis progression, either stabilising or even decreasing…

Unmet needs in children with FH

Identification and treatment to reduce low density lipoprotein cholesterol (LDL-C) of children with familial hypercholesterolaemia (FH) is vital to reduce the risk of cardiovascular death in early adulthood. FH is the most common genetic disorder in the world. Parents and healthcare professionals need to be…

Improving the management of the FH patient

Professor Raul Santos from Brazil says that the new therapies which inhibit PCSK9 to significantly reduce low density Lipoprotein Cholesterol (LDL-C) when given in addition to other cholesterol lowering drugs offer a very important advance in reducing cardiovascular risk in people with Familial Hypercholesterolemia (FH).

Do the PCSK9 monoclonal antibodies have neurocognitive adverse effects?

Evolocumab

ACC 2014 March 29th  MENDEL-2: http://www.pcsk9forum.org/mendel-2-2/ DESCARTES: http://www.pcsk9forum.org/descartes-durable-ldl-c-lowering-with-evolocumab/ RUTHERFORD-2: http://www.pcsk9forum.org/acc-2014-rutherford-2-evolocumab-in-heterozygous-fh/ LAPLACE-2: http://www.pcsk9forum.org/acc-2014-latebreaker-laplace-2-evolocumb-effective-in-patients-on-high-and-moderate-intensity-statins/ GAUSS-2: http://www.pcsk9forum.org/acc-2014-latebreaker-gauss-2-evolocumab-addresses-unmet-needs-in-statin-intolerance/  

Lipoprotein apheresis lowers plasma PCSK9

Lipoprotein apheresis is clearly important in the management of severe familial hyperchoelsterolaemia (FH). However, its use is not without practical and cost limitations. Evidence that lipoprotein apheresis has an additional benefit in lowering plasma PCSK9 levels, beyond effects on low-density lipoprotein cholesterol (LDL-C), suggests that…

PCSK9: From discovery to therapeutic applications

PUBMED abstract: http://www.ncbi.nlm.nih.gov/pubmed/24373748

TESLA in brief

TESLA was a randomised, double-blind, placebo-controlled trial of the PCSK9 inhibitor, evolocumab, in 50 patients (49 received treatment) with homozygous FH (mean LDL-C at baseline 9.0 mmol/L). All were on statins and 91% also received ezetimibe. LDL receptor mutations were identified in 45 (92%) patients;…

How low should LDL cholesterol be lowered – and for how long?

RUTHERFORD-2 in brief

RUTHERFORD-2 was a large, randomised, placebo-controlled trial of the PCSK9 inhibitor, evolocumab, in 331 patients with heterozygous familial hypercholesterolaemia (mean LDL-C at baseline 3.9-4.2 mmol/L). All were on statins and 62% were also on ezetimibe. FH-causing mutations were identified in 80% of patients, most were…