Treatment Overview

Living with familial hypertriglyceridemia can feel like an uphill battle. Because this condition is genetic, many people find that even strict dieting and regular exercise are not enough to bring their triglyceride levels down to a healthy range. It is often a “silent” condition, discovered only through routine blood work, yet the risk it poses to long-term health is real. For many, the diagnosis brings a mix of confusion and anxiety about how to manage a condition that is written into their DNA.

Treatment is critical because extremely high triglyceride levels thicken the blood, significantly increasing the risk of pancreatitis, a painful and potentially dangerous inflammation of the pancreas. Furthermore, long-term elevation contributes to hardening of the arteries and heart disease. Treatment plans are highly personalized; what works for a patient with moderately high levels may differ from the aggressive therapy needed for someone with severe elevations. The goal is to protect the pancreas and cardiovascular system by keeping lipid levels in check (National Human Genome Research Institute, 2013).

Overview of treatment options for Familial Hypertriglyceridemia

The primary objective of treatment is to lower serum triglyceride levels to reduce the risk of acute pancreatitis and cardiovascular events. While lifestyle modifications such as limiting alcohol, reducing sugar intake, and losing weight are the foundation of care, the genetic nature of this condition means that medications are almost always necessary.

For mild cases, doctors may emphasize diet changes first. However, for familial hypertriglyceridemia, where levels can be persistently high, pharmacological intervention is standard. The approach often involves a combination of therapies targeting different aspects of lipid metabolism. Treatment is generally chronic and lifelong, requiring consistent management rather than a short-term cure.

Medications used for Familial Hypertriglyceridemia

Fibrates are widely considered the first-line medication for treating severe hypertriglyceridemia. Drugs such as fenofibrate and gemfibrozil are specifically designed to target triglycerides. Clinical experience suggests that fibrates can lower triglyceride levels by 25% to 50%, making them a powerful tool for preventing pancreatitis in high-risk patients.

Prescription-strength omega-3 fatty acids are another common treatment. Unlike over-the-counter fish oil supplements, which vary in potency, prescription formulations (such as icosapent ethyl or omega-3-acid ethyl esters) provide a concentrated dose regulated by the FDA. These are often used alongside other medications to help drive numbers down further.

Statins, while primarily known for lowering LDL (bad) cholesterol, are also frequently prescribed. Medications like atorvastatin or rosuvastatin have a modest effect on triglycerides but are crucial for reducing the overall risk of heart attacks and strokes, which is elevated in these patients. In some cases, niacin (vitamin B3) may be used, though it is less common today due to side effects. Patients can typically expect to see improvements in their lipid panel within a few weeks of starting these therapies (American Heart Association, 2020).

How these medications work

Fibrates work by activating a specific protein in the body that speeds up the breakdown of triglyceride-rich particles in the blood. They essentially help the body clear fats from the bloodstream more efficiently.

Prescription omega-3 fatty acids primarily target the liver. They inhibit the liver’s production of triglycerides and VLDL (very-low-density lipoprotein) carriers. By reducing the amount of fat the factory (the liver) sends out into the bloodstream, overall levels decrease.

Statins work by blocking a substance the liver needs to make cholesterol. While their main target is cholesterol, this process also helps the liver clear lipoproteins from the blood, providing a secondary benefit of lowering triglycerides and stabilizing the health of blood vessel walls.

Side effects and safety considerations

While these medications are generally safe, they do carry risks. Fibrates and statins can cause muscle pain; combining them slightly increases the risk of muscle damage, necessitating close monitoring. Prescription omega-3s often cause fishy aftertaste, upset stomach, or prolonged bleeding.

Liver function is a major safety concern; both fibrates and statins affect the liver, requiring routine enzyme monitoring. They are usually avoided in pregnancy. Patients should seek immediate care for severe, unexplained muscle pain, dark urine, or severe abdominal pain, as these may signal serious adverse reactions (MedlinePlus, 2022).

Since everyone’s experience with the condition and its treatments can vary, working closely with a qualified healthcare provider helps ensure safe and effective care.

References

  1. American Heart Association. https://www.heart.org
  2. MedlinePlus. https://medlineplus.gov
  3. National Human Genome Research Institute. https://www.genome.gov
  4. National Heart, Lung, and Blood Institute. https://www.nhlbi.nih.gov

Medications for Familial Hypertriglyceridemia

These are drugs that have been approved by the US Food and Drug Administration (FDA), meaning they have been determined to be safe and effective for use in Familial Hypertriglyceridemia.

Found 9 Approved Drugs for Familial Hypertriglyceridemia

Fenofibrate

Brand Names
Tricor, Lipofen

Fenofibrate

Brand Names
Tricor, Lipofen
Form: Tablet, Capsule
Method of administration: Oral
FDA approval date: April 09, 2002
Classification: Peroxisome Proliferator Receptor alpha Agonist
TRICOR is a peroxisome proliferator-activated receptor (PPAR) alpha agonist indicated as an adjunct to diet: To reduce elevated LDL-C, Total-C, TG and Apo B, and to increase HDL-C in adult patients with primary hypercholesterolemia or mixed dyslipidemia.

Lopid

Generic Name
Gemfibrozil

Lopid

Generic Name
Gemfibrozil
Form: Tablet
Method of administration: Oral
FDA approval date: September 27, 1993
Classification: Peroxisome Proliferator Receptor alpha Agonist
Gemfibrozil tablets, USP are indicated as adjunctive therapy to diet for: 1. Treatment of adult patients with very high elevations of serum triglyceride levels (Types IV and V hyperlipidemia) who present a risk of pancreatitis and who do not respond adequately to a determined dietary effort to control them. Patients who present such risk typically have serum triglycerides over 2000 mg/dL and have elevations of VLDL-cholesterol as well as fasting chylomicrons (Type V hyperlipidemia). Subjects who consistently have total serum or plasma triglycerides below 1000 mg/dL are unlikely to present a risk of pancreatitis. Gemfibrozil therapy may be considered for those subjects with triglyceride elevations between 1000 and 2000 mg/dL who have a history of pancreatitis or of recurrent abdominal pain typical of pancreatitis. It is recognized that some Type IV patients with triglycerides under 1000 mg/dL may, through dietary or alcoholic indiscretion, convert to a Type V pattern with massive triglyceride elevations accompanying fasting chylomicronemia, but the influence of Gemfibrozil therapy on the risk of pancreatitis in such situations has not been adequately studied. Drug therapy is not indicated for patients with Type I hyperlipoproteinemia, who have elevations of chylomicrons and plasma triglycerides, but who have normal levels of very low density lipoprotein (VLDL). Inspection of plasma refrigerated for 14 hours is helpful in distinguishing Types I, IV, and V hyperlipoproteinemia. 2. Reducing the risk of developing coronary heart disease only in Type IIb patients without history of or symptoms of existing coronary heart disease who have had an inadequate response to weight loss, dietary therapy, exercise, and other pharmacologic agents (such as bile acid sequestrants and nicotinic acid, known to reduce LDL-and raise HDL-cholesterol) and who have the following triad of lipid abnormalities: low HDL-cholesterol levels in addition to elevated LDL-cholesterol and elevated triglycerides. The National Cholesterol Education Program has defined a serum HDL-cholesterol value that is consistently below 35 mg/dL as constituting an independent risk factor for coronary heart disease. Patients with significantly elevated triglycerides should be closely observed when treated with gemfibrozil. In some patients with high triglyceride levels, treatment with gemfibrozil is associated with a significant increase in LDL-cholesterol. BECAUSE OF POTENTIAL TOXICITY SUCH AS MALIGNANCY, GALLBLADDER DISEASE, ABDOMINAL PAIN LEADING TO APPENDECTOMY AND OTHER ABDOMINAL SURGERIES, AN INCREASED INCIDENCE IN NON-CORONARY MORTALITY, AND THE 44% RELATIVE INCREASE DURING THE TRIAL PERIOD IN AGE-ADJUSTED ALL-CAUSE MORTALITY SEEN WITH THE CHEMICALLY AND PHARMACOLOGICALLY RELATED DRUG, CLOFIBRATE, THE POTENTIAL BENEFIT OF GEMFIBROZIL IN TREATING TYPE IIA PATIENTS WITH ELEVATIONS OF LDL-CHOLESTEROL ONLY IS NOT LIKELY TO OUTWEIGH THE RISKS. GEMFIBROZIL IS ALSO NOT INDICATED FOR THE TREATMENT OF PATIENTS WITH LOW HDL-CHOLESTEROLAS THEIR ONLY LIPID ABNORMALITY. In a subgroup analysis of patients in the Helsinki Heart Study with above-median HDL-cholesterol values at baseline (greater than 4.

Atorvastatin

Brand Names
Lipitor, Lotrel, Katerzia, Amlodipine, Benazepril, Amlodipine Besylate, Azor, Caduet, Norliqva, Atorvaliq, Lotensin, Olmesartan Medoxomil, Benicar, Tribenzor, Olmesartan Medoxomil Amlodipine, Norvasc

Atorvastatin

Brand Names
Lipitor, Lotrel, Katerzia, Amlodipine, Benazepril, Amlodipine Besylate, Azor, Caduet, Norliqva, Atorvaliq, Lotensin, Olmesartan Medoxomil, Benicar, Tribenzor, Olmesartan Medoxomil Amlodipine, Norvasc
Form: Tablet, Suspension, Capsule, Solution
Method of administration: Oral
FDA approval date: June 21, 1991
Classification: HMG-CoA Reductase Inhibitor
Atorvastatin calcium tablets are indicated: To reduce the risk of: Myocardial infarction (MI), stroke, revascularization procedures, and angina in adults with multiple risk factors for coronary heart disease (CHD) but without clinically evident CHD MI and stroke in adults with type 2 diabetes mellitus with multiple risk factors for CHD but without clinically evident CHD Non-fatal MI, fatal and non-fatal stroke, revascularization procedures, hospitalization for congestive heart failure, and angina in adults with clinically evident CHD As an adjunct to diet to reduce low-density lipoprotein cholesterol (LDL-C) in: Adults with primary hyperlipidemia. Adults and pediatric patients aged 10 years and older with heterozygous familial hypercholesterolemia (HeFH). As an adjunct to other LDL-C-lowering therapies, or alone if such treatments are unavailable, to reduce LDL-C in adults and pediatric patients aged 10 years and older with homozygous familial hypercholesterolemia (HoFH). As an adjunct to diet for the treatment of adults with: Primary dysbetalipoproteinemia Hypertriglyceridemia Atorvastatin calcium is an HMG-CoA reductase inhibitor (statin) indicated ( 1 ): To reduce the risk of: Myocardial infarction (MI), stroke, revascularization procedures, and angina in adults with multiple risk factors for coronary heart disease (CHD) but without clinically evident CHD. MI and stroke in adults with type 2 diabetes mellitus with multiple risk factors for CHD but without clinically evident CHD. Non-fatal MI, fatal and non-fatal stroke, revascularization procedures, hospitalization for congestive heart failure, and angina in adults with clinically evident CHD. As an adjunct to diet to reduce low-density lipoprotein (LDL-C) in: Adults with primary hyperlipidemia. Adults and pediatric patients aged 10 years and older with heterozygous familial hypercholesterolemia (HeFH). As an adjunct to other LDL-C-lowering therapies to reduce LDL-C in adults and pediatric patients aged 10 years and older with homozygous familial hypercholesterolemia. As an adjunct to diet for the treatment of adults with: Primary dysbetalipoproteinemia. Hypertriglyceridemia.

Altoprev

Generic Name
Lovastatin

Altoprev

Generic Name
Lovastatin
Form: Tablet
Method of administration: Oral
FDA approval date: December 17, 2001
Classification: HMG-CoA Reductase Inhibitor
Therapy with lovastatin tablets USP should be a component of multiple risk factor intervention in those individuals with dyslipidemia at risk for atherosclerotic vascular disease. Lovastatin tablets USP should be used in addition to a diet restricted in saturated fat and cholesterol as part of a treatment strategy to lower total-C and LDL-C to target levels when the response to diet and other nonpharmacological measures alone has been inadequate to reduce risk. Primary Prevention of Coronary Heart Disease In individuals without symptomatic cardiovascular disease, average to moderately elevated total-C and LDL-C and below average HDL-C, lovastatin tablets USP are indicated to reduce the risk of: -Myocardial infarction -Unstable angina -Coronary revascularization procedures (See CLINICAL PHARMACOLOGY, Clinical Studies). Coronary Heart Disease Lovastatin tablets USP are indicated to slow the progression of coronary atherosclerosis in patients with coronary heart disease as part of a treatment strategy to lower total-C and LDL-C to target levels. Hypercholesterolemia Therapy with lipid-altering agents should be a component of multiple risk factor intervention in those individuals at significantly increased risk for atherosclerotic vascular disease due to hypercholesterolemia. Lovastatin tablets USP are indicated as an adjunct to diet for the reduction of elevated total-C and LDL-C levels in patients with primary hypercholesterolemia (Types IIa and IIb 2 ), when the response to diet restricted in saturated fat and cholesterol and to other nonpharmacological measures alone has been inadequate. 2 Classification of Hyperlipoproteinemias IDL = intermediate-density lipoprotein. Type Lipoproteins elevated Lipid Elevations major minor I chylomicrons TG ↑→C IIa LDL C — IIb LDL,VLDL C TG III (rare) IDL C/TG — IV VLDL TG ↑→C V (rare) chylomicrons,VLDL TG ↑→C Adolescent Patients with Heterozygous Familial Hypercholesterolemia Lovastatin tablets USP are indicated as an adjunct to diet to reduce total-C, LDL-C and apolipoprotein B levels in adolescent boys and girls who are at least one year post-menarche, 10 to 17 years of age, with heFH if after an adequate trial of diet therapy the following findings are present: 1. LDL-C remains >189 mg/dL or 2. LDL-C remains >160 mg/dL and: there is a positive family history of premature cardiovascular disease or two or more other CVD risk factors are present in the adolescent patient General Recommendations Prior to initiating therapy with lovastatin, secondary causes for hypercholesterolemia (e.g., poorly controlled diabetes mellitus, hypothyroidism, nephrotic syndrome, dysproteinemias, obstructive liver disease, other drug therapy, alcoholism) should be excluded, and a lipid profile performed to measure total-C, HDL-C, and TG. For patients with TG less than 400 mg / dL.

Omega-3-Acid

Generic Name
Omega-3-Acid

Omega-3-Acid

Generic Name
Omega-3-Acid
Form: Capsule
Method of administration: Oral
FDA approval date: April 07, 2014
Classification: Omega-3 Fatty Acid
Omega-3-acid ethyl esters capsules are indicated as an adjunct to diet to reduce triglyceride (TG) levels in adult patients with severe (greater than or equal to 500 mg/dL) hypertriglyceridemia. Usage Considerations: Patients should be placed on an appropriate lipid-lowering diet before receiving omega-3-acid ethyl esters capsules and should continue this diet during treatment with omega-3-acid ethyl esters capsules. Laboratory studies should be done to ascertain that the lipid levels are consistently abnormal before instituting therapy with omega-3-acid ethyl esters capsules. Every attempt should be made to control serum lipids with appropriate diet, exercise, weight loss in obese patients, and control of any medical problems such as diabetes mellitus and hypothyroidism that are contributing to the lipid abnormalities. Medications known to exacerbate hypertriglyceridemia (such as beta blockers, thiazides, estrogens) should be discontinued or changed, if possible, prior to consideration of TG-lowering drug therapy. Limitations of Use: The effect of omega-3-acid ethyl esters capsules on the risk for pancreatitis has not been determined. The effect of omega-3-acid ethyl esters capsules on cardiovascular mortality and morbidity has not been determined. Omega-3-acid ethyl esters capsules are a combination of ethyl esters of omega 3 fatty acids, principally eicosapentaenoic acid (EPA) and docosahexaenoic acid (DHA), indicated as an adjunct to diet to reduce triglyceride (TG) levels in adult patients with severe (≥500 mg/dL) hypertriglyceridemia. Limitations of Use: The effect of omega-3-acid ethyl esters capsules on the risk for pancreatitis has not been determined. The effect of omega-3-acid ethyl esters capsules on cardiovascular mortality and morbidity has not been determined.
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