In one study, about 80% of patients with familial hypercholesterolemia who received a statin did not develop CHD, compared to slightly less than 40% of those who didn't get a statin.
Statins can be used in children as young as age 8, or even earlier in homozygous FH.6 While a physician might be hesitant to start a chronic medication in a young patient, research shows that earlier intervention results in additional years of life.23 To date, no significant adverse effects of statins in pediatric patients have been identified, and statins have not been shown to impair growth.24,25 Young female patients should be counseled about the adverse effects statins can have on a fetus if the patient becomes pregnant while taking the medication.
Navigating the waters of statin treatment
Musculoskeletal symptoms are the most common adverse effect reported by patients taking statins. A thorough assessment of a patient’s muscle complaints is necessary to avoid prematurely concluding that he or she cannot tolerate statins.
A study in which “statin-intolerant” patients were re-challenged found that more than 90% of patients could tolerate statins through the course of the one-year study and that it was likely that the patients’ initial muscle complaints were not due to statin use.23 (To read more about potential adverse events of statins, see “Statin adverse effects: Sorting out the evidence,” J Fam Pract. 2014;63:497-506.).
The goal of treatment for familial hypercholesterolemia is to reduce LDL levels by 50%.
If LDL levels in a patient with HeFH remain at or above 160 mg/dL, intensifying treatment by adding another lipid-lowering medication might be warranted.22 For patients with HoFH, in whom the condition is more quickly life-threatening, there are additional choices, including LDL apheresis and medications such as mipomersen and lomitapide. Both of these medications can cause hepatotoxicity, and are available only through a Risk Evaluation and Mitigation Strategy program, which means they can only be prescribed by certified physicians. PCSK9 inhibitors are in the pipeline and may one day help patients with HoFH by addressing one of the genetic causes of this disorder.
