Skip to main content

New hope treatment in familial hypercholesterolemia

New hope comes today for those with familial hypercholesterolemia (FH).

Familial hypercholesterolemia (FH) is a genetic disorder characterized by high cholesterol levels, specifically very high levels of low-density lipoprotein (LDL, "bad cholesterol"), in the blood and early cardiovascular disease.



Without treatment, the life expectancy of those with familial hypercholesterolemia can be reduced by approximately 15-30 years. However, in people with homozygous familial hypercholesterolemia, life expectancy may only be 20 years or less.

Homologous chromosomes come in pairs. One homologous chromosome is inherited from the mother; the other is inherited from the father.  Homozygous refers to a gene that has identical alleles on both homologous chromosomes.   This is why the homozygous condition is particularly rare and can be more severe.

HoFH is a rare and more severe form of familial hypercholesterolemia (FH), an inherited condition characterized by very high levels of LDL-C and premature cardiovascular disease.

Inheriting FH- causing mutations from both parents results in HoFH in the child. HoFH is an extremely severe condition and is often associated with aggressive and markedly premature heart disease, including coronary artery disease and aortic valve disease. 

Children and teenagers with HoFH may have heart attacks or angina requiring the insertion of coronary stents or heart bypass surgery.

A new study shows that adding Praluent® (alirocumab) significantly reduced cholesterol levels by 35.6 per cent, compared to other lipid-lowering therapies alone, in patients with homozygous familial hypercholesterolemia.

The results of the placebo-controlled trial were presented at the joint meeting of the American College of Cardiology (ACC.20) together with the World Congress of Cardiology (WCC) Scientific Sessions.

Results showed treatment with Praluent significantly reduced other lipoprotein and lipid measures associated with elevated cardiovascular risk, including apolipoprotein B [29.8 percent (p<0.0001)], total cholesterol [26.5 percent (p<0.0001)], non-high-density lipoprotein cholesterol (HDL-C) [32.9 percent (p<0.0001)] and lipoprotein(a) [28.4 percent (p<0.0001)] versus placebo.

Lead investigator Dr Dirk Blom says,

“These results demonstrate a statistically significant and clinically-meaningful reduction in LDL-C levels after 12 weeks and have the potential to expand treatment options for very high-risk patients with this serious condition.”



Comments

Popular posts from this blog

The lion and the wildebeest

Birds flock, fish school, bees swarm, but social being is more than simply sticking together.  Social groups enable specialisation and a sharing of abilities, and enhances ability, learning and creating new tricks. The more a group works together, the more effective they become as a team.  Chimpanzees learn from each other how to use stones to crack nuts, or sticks to get termites.  All around us we see cooperation and learning in nature.  Nature is inherently creative.  Pulling together becomes a rallying cry during a crisis.  We have heard it throughout the coronavirus pandemic.  "We are all in this together", a mantra that encourages people to adopt a common strategy. In an era of 'self-interest' and 'survival of the fittest,'  and 'selfish gene', we lose sight of the obvious conclusion from the evidence all around us.   Sticking together is more often the better approach.  This is valid for the lion as it is also for the wildebeest.   We don't

Noise pollution puts nature at risk

 "I just want a bit of peace and quiet!" Let's get away from all the hustle and bustle; the sound of endless traffic on the roads, of the trains on the railway, and the planes in the sky; the incessant drone; the noise. We live in a world of man-made noise; screeching, bellowing, on-and-on in an unmelodious cacophony.  This constant background noise has now become a significant health hazard.   With average background levels of 60 decibels, those who live in cities are often exposed to noise over 85 decibels, enough to cause significant hearing loss over time.  It causes stress, high blood pressure, headache and loss of sleep and poor health and well-being.   In nature, noise has content and significance.  From the roar of the lion, the laughing of a hyena,  communication is essential for life; as the warning of danger, for bonding as a group or a pair, finding a mate, or for establishing a position in a hierarchy - chattering works.  Staying in touch is vital to working

Therapeutic animal stress

Interacting with animals is known to be therapeutic,  particularly in reducing stress.  But do we consider sufficiently the effects this may have on the animals involved?   We might assume that because it is calming for us, then it must be so for the therapeutic animals, but is this so?  New research suggests that it isn't always without stress for the animals involved.  Positive human-animal interaction relates to changes in physiological variables both in humans and other animals, including a reduction of subjective psychological stress (fear, anxiety) and an increase of oxytocin levels in the brain.  It also reduces the 'stress' hormone, cortisol. Indeed, these biological responses have measurable clinical benefits.  Oxytocin has long been implicated in maternal bonding, sexual behaviour and social affiliation behaviours and in promoting a sense of well-being .  So far, so good.  We humans often turn to animals for stress relief, companionship, and even therapy.  We kno