Which medications are used in the treatment of pediatric lipid disorders?

Updated: Jun 27, 2019
  • Author: Henry J Rohrs, III, MD; Chief Editor: Stuart Berger, MD  more...
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Answer

Statins are the initial drugs of choice for the treatment of type IIA HLP in children. Statins are safe and highly effective. As a result of a lack of adverse effects, compliance is usually high with the use for statins. Lovastatin, simvastatin, atorvastatin, and pravastatin appear to be equally efficacious and safe in children.

Bile acid–binding resins are safe because they are not systemically absorbed and typically do not produce renal toxicity or hepatotoxicity. However, these drugs are not typically palatable; therefore, compliance is usually poor and prevents their widespread and long-term use in children with type IIA HLP. Bile acid–binding resins do not reduce LDL-C levels as effectively as statins do.

Niacin is useful in various phenotypes (eg, HLP types IIA, IIB, or IV), although LDL-C levels are not lowered as effectively as through the use of statins. Flushing and GI tract upset usually interfere with long-term compliance with niacin. In addition, niacin is likely to display hepatotoxicity equal to that of statins.

The primary use of gemfibrozil is in the treatment of HLP types IIA, IIB, IV or V. In adults, this drug is usually safe and effective.

When treating children with type IIA HLP, the authors believe that it is prudent to discuss the advantages and disadvantages of each agent with the patient’s parents. See the diagram summary below.

Pediatric lipid disorders in clinical practice. Ph Pediatric lipid disorders in clinical practice. Pharmacologic approach to the treatment of type IIA hyperlipoproteinemia (HLP).

In patients with type IIB HLP, a statin would be the initial drug of choice. Physicians should avoid the use of bile acid–binding resins in patients with type IIB HLP because resin therapy can worsen hypertriglyceridemia. See the image below.

Pediatric lipid disorders in clinical practice. Ph Pediatric lipid disorders in clinical practice. Pharmacologic approach to the treatment of type IIB hyperlipoproteinemia (HLP).

Treatment of isolated or predominant hypertriglyceridemia (type IV phenotype) is controversial. Niacin is the drug of choice. See the diagram below.

Pediatric lipid disorders in clinical practice. Ph Pediatric lipid disorders in clinical practice. Pharmacologic approach to the treatment of type IV hyperlipoproteinemia (HLP).

Gemfibrozil can be administered if niacin is ineffective or produces unacceptable adverse effects. Because an increasing number of children are recognized as being at risk for premature cardiovascular disease, the authors believe that studies of the safety and efficacy of lipid-lowering drugs in children should be greatly expanded.


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