Abstract Body: Background: Randomized clinical trials show short- and medium-term safety and good LDL-C lowering of lipid lowering therapy (LLT) in pediatric patients, with 40-60% of patients reaching their LDL-C goals. However, there is a paucity of data on effectiveness of LLT in real-world pediatric practice. We aimed to describe the proportion of pediatric patients treated with LLT at our center who reached LDL-C goal (<130 mg/dL or <100 mg/dL in high-risk patients), and to identify patient characteristics associated with reaching goal LDL-C levels.
Methods: This single center retrospective study reviews patients on LLT cared for in a pediatric preventive cardiology program between 2010 and 2024. The percentage of patients who reach goal LDL-C at least once within one year of statin initiation was described. The cumulative time weighted LDL was estimated using the area under the LDL-C vs. age trajectory, expressed as “mg/dL years”. We also compared baseline patient characteristics (including usual vs. high-risk) for patients who reached goal LDL-C versus those who did not.
Results: Of 1,307 patients evaluated for dyslipidemia during this timeframe, 719 were prescribed a statin and 628 had a baseline LDL-C; 322 patients on statin therapy that had a follow-up LDL-C within one year of statin initiation were included. Mean age of patients was 14.2 years (SD 3.86 years) and 47% were female. Patients were primarily treated with atorvastatin (70%) and simvastatin (25%). Overall, 69% of patients reached goal LDL-C levels within one year of statin initiation. Of those who were evaluated and reached goal, 66% were usual risk and 3% were high risk. Baseline characteristics significantly associated with reaching goal LDL-C included higher SBP, older age, lower LDL-C at baseline, lower total cholesterol at baseline, lower HbA1C at baseline, lower first-year cumulative time weighted LDL and lower first-year mean time weighted LDL-C.
Conclusions: A majority of patients were able to reach goal LDL-C within one year of starting LLT, however, 31% did not reach goal. Attention to younger patients with high LDL-C may be necessary to help patients with hypercholesterolemia reach their goals. Lower LDL-C at baseline was associated with reaching goal, which suggests that for patients with higher LDL-C either higher doses or multiple drug therapy might be indicated.