Burden of extremely high triglycerides

Extremely high triglycerides can persist despite standard of care.

Traditional treatment options are limited

Medicines typically used to lower triglycerides, such as statins, fibrates, and omega-3 fatty acids, may not suffice for people with extremely high triglycerides, including people with familial chylomicronemia syndrome (FCS).

Several triglyceride-lowering therapies that are used to manage triglycerides have limited efficacy for people with FCS or with diseases with a similar presentation. Most traditional triglyceride-lowering therapies activate lipoprotein lipase (LPL) activity, which is missing or nonfunctional in people with FCS.

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Statins

Statins may be useful in protecting against atherosclerotic cardiovascular disease (ASCVD); however, they are not clinically useful for people with FCS and do not lower acute pancreatitis risk.
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Fibrates

Fibrates induce triglyceride clearance by activating LPL activity; however, due to the loss of LPL activity in people with FCS, the clinical benefit of fibrates is minimal.
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Eicosapentaenoic Acid (EPA)

EPA, an omega-3 fatty acid, has been shown to moderately reduce triglyceride levels caused by other diseases but are not effective for people with FCS.

Restrictive diets negatively impact quality of life

Some people use intensive dieting to manage high triglycerides, however this strategy that avoids ingesting fats to maintain lower overall fasting triglycerides is very burdensome.

People with extremely high triglycerides that persist despite standard of care may try to prevent rises in triglyceride levels through dietary restriction of total fat intake, abstinence from alcohol, and avoidance of medications known to increase triglyceride levels.

In a survey of a 166 people with FCS, participants reported their restrictive diet was:

Extremely time consuming

Energy draining

Ineffective at stopping all symptoms

Even with treatment, extremely
high triglycerides take an emotional and social toll

People with FCS report that their disease impacts their mental well-being and their social lives.

In a survey of 166 people with FCS, the most common emotional symptoms were:

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Constant uncertainty about the possibility of acute pancreatitis or pain.
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Anxiety, fear, or worry about their health.
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Uncertainty about what or how much to eat.
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Feeling out of control or helpless because of their FCS.

In a survey of 166 people with FCS, participants reported their disease negatively impacted: 

Employment

Emotional and mental well-being

Social relationships

An online, anonymous, quantitative survey of people with FCS from 10 countries (Australia, Canada, Germany, India, Netherlands, Portugal, Spain, Sweden, United Kingdom, and United States). Results were patient reported.

Extremely high tryglycerides have systemic consequences

In addition to causing acute pancreatitis, FCS can impact the liver, spleen, heart, and eyes, and cause other systemic problems.

Symptoms and comorbidities reported in a survey of 166 people with FCS:

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  • Difficulty concentrating (16%)
  • Impaired judgment (11%)
  • Lipemia retinalis (9%)
  • Brain fog (8%)
  • Forgetfulness (8%)

We’ll get there [soon]

Imagine if we could reduce the risk of acute pancreatitis

Guidelines consistently establish <500 mg/dL as the goal of triglyceride management.

See the risk

siRNA may hold the key to managing extremely high triglycerides

Silencing genes with siRNA (small interfering RNA) could be in our future.

See how siRNA works

Show References
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