Background: There is limited research detailing LDL trends over the long term in children on various lipid lowering medications.
Objectives: This study sought to assess factors associated with stability of LDL levels in children on long-term pharmacotherapy and their ability to reach the current recommended LDL goal of 130 mg/dL or less while on pharmacotherapy.
Methods: Medical records of children seen in a university pediatric cholesterol clinic between 1998 and 2012 who were treated with a statin, ezetimibe, or both were reviewed. Aggregate data were obtained to determine the number of children who were able to reach an LDL level of 130 mg/dL or less while on pharmacotherapy. Kaplan-Meier curve and proportional hazard regression analysis were used to examine the propensity for LDL levels to stabilize over time while on pharmacotherapy as well as factors affecting this propensity.
Results: 76 patients who contributed 864 total visits met inclusion criteria. 56 of 76 patients developed a stable LDL with median time to stability of 28 months on pharmacotherapy. Younger age of first visit was associated with development of LDL stability (9.8 years for stable LDL vs. 13.3 years for nonstable LDL; p= 0.004). Titration to higher statin doses and/or higher potency statins was also associated with higher propensity for LDL stabilization. There was no significant difference between patients who were taking a statin, ezetimibe, or both in terms of their propensity to stabilize even when stratifying for presence of probable familial hypercholesterolemia (p=0.579). 36 of 76 patients were able to reach an LDL of 130 mg/dL or less, with only 11 of 38 patients with probable familial hypercholesterolemia (>190 mg/dL baseline LDL) reaching this goal.
Conclusion: A majority of children in a university pediatric cholesterol clinic were able to reach a stable LDL level on pharmacotherapy after a median 28 month interval. On the other hand, the majority of children had difficulty reaching the current recommended goal LDL of 130 mg/dL or less, even after aggressive titration of medication. This was specifically true for those with probable familial hypercholesterolemia. Further research is needed to assess the utility of goal-directed therapy in children with regards to cardiovascular outcomes.