Gidding - Figure 14 - Treatment rationale Text

In summary, the treatment rationale includes:

• begin treatment above age 8-10 years in anybody with serum LDL cholesterol >190 mg/dL or >160 mg/dL after diet intervention (or consider treatment at >160 mg/dL if there are multiple risk factors present);
• clinical trials of statin therapy with medium-term follow-up suggest that both safety and efficacy are optimal with this therapy, although long-term data and studies are still lacking;
• a goal for young FH patients is to try to achieve a 50% reduction, or an LDL level <130 mg/dL.

One important difference between pediatric and adult treatment is the need to assess the balance between increased dosing and the potential for long-term side effects over many years. We know that statins do not have a direct linear response in terms of LDL lowering, so there is a lot of lowering with a small dose of statin, but the dose increases the percent lowering goes down. Therefore it is necessary to ask the question:

• Is it worth trying to get to a target goal but to have to double or quadruple the statin exposure for decades of life?
• Or, at a very early stage of atherosclerosis development, simply go for the 50% lowering with intensification of treatment at later ages.

Gidding S. J Clin Lipidol. 2011; 5(6).