CMFT researchers set to revolutionise way high cholesterol is managed through Heart UK strategy
The Heart UK consensus statement, led by the Manchester lipid clinic and lipoprotein services team at Central Manchester Hospitals, published in the journal Atherosclerosis, is set to revolutionise care for patients with homozygous familial hypercholesterolaemia (HoFH).
The statement sets out a strategy for combining treatment with cholesterol lowering drugs , lipoprotein apheresis (removal of cholesterol from the bloodstream) and liver transplantation as a last resort, to reduce cholesterol levels in these patients. It also provides doctors with the latest guidance on the clinical management of pregnancy and contraception.
FH is a genetic condition characterised by high cholesterol levels in the blood. It is due to mutations affecting the function of the LDL receptor, which is a protein designed to remove bad (LDL) cholesterol from the circulation.
Those with one mutation, heterozygous FH, are likely to develop premature coronary artery disease. Typically men develop heart disease in their 50s and women in their 60s if not treated, but statin drugs are very effective.
Those with homozygous FH have two such mutations. People with the condition often experience heart disease in childhood and may not respond to medical therapy to reduce cholesterol levels. The clinical manifestations of HoFH are not only more severe than in HeFH but there are also unique features such as early damage to heart valves. The statement recommends the re-classification of HoFH as a rare disease within the UK strategy for rare diseases, which is important for funding treatment.
HoFH is rare, affecting at least 1 in a million births but genetic diagnosis suggests that it may be twice as common. It, nevertheless, represents a significant burden for affected families and the health service.
The new strategy aims to achieve lower cholesterol levels for patients receiving treatment for homozygous FH, in line with those for the general FH population to improve cardiovascular outcomes.
The new statement translates the latest research findings from the Cardiovascular Trials Unit at CMFT and other institutions into clinical practice, to standardise and improve patients care.
In developing the statement, clinicians reviewed the effectiveness of current types of treatment for homozygous FH:
- Cholesterol lowering drugs in HoFH
- Lipoprotein apheresis – a process resembling dialysis, where the cholesterol containing particles are removed from the bloodstream
- Liver transplantation
The lead authors, Dr Mike France and Dr Handrean Soran (chair of the Medical, Scientific and research committee of HEART UK) played important roles in the development of the statement.
Dr Mike France, Consultant Chemical Pathologist says: “lowering cholesterol in children with HoFH to internationally agreed levels has the potential to normalise life expectancy in this otherwise lethal condition.”
Dr Handrean Soran, Consultant Physician & Endocrinologist at CMFT/Honorary Senior Lecturer at The University of Manchester adds:
“This statement brings together the latest research and best practice in the management of cholesterol levels in patients with homozygous FH. We hope that it will help clinicians in the UK and beyond to make more informed decisions about how to support patients in managing their condition.
“Our aim is to reduce and maintain low levels of bad cholesterol in these patients, and in turn reduce the need for liver transplantation and the incidence of cardiovascular events.”
