SUBOPTIMAL ADHERENCE TO STATIN THERAPY IN CHILDREN AND ADOLESCENTS WITH HETEROZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA DESPITE A HIGH THERAPEUTIC EFFICACY: IS THE CARDIOVASCULAR RISK UNDERESTIMATED?

Background. European guidelines currently support the initiation of statin by age 8-10 years in patients with heterozygous familial hypercholesterolemia (HeFH) to slow the progression of endothelial disfunction and to reduce the risk of cardiovascular disease in adulthood. However, to date, there is lack of data on adherence to statins in the paediatric population. Therefore, we describe our real-life paediatric experience about efficacy and adherence of statin therapy. Methods. This is a monocentric, observational study recruiting children and adolescents with genetically confirmed HeFH. Anthropometric measures, fasting lipid profile and therapeutic data were collected at diagnosis of HeFH [T0], on lipid-lowering diet [T1], four weeks after starting statin therapy [T2] and yearly during the first two years on statin [T3 and T4]. Results. 24 HeFH children and adolescents (17/24 female) were started on statin at a mean age of 13.77±3.09 years (12 on atorvastatin, 10 on pravastatin and 2 on simvastatin). On lipid-lowering diet, lipid metabolism did not change significantly [LDL-C 237.61±47.18 vs. 218.22±50.15 mg/dl, p 0.11], while on statin the improvement was quick and persistent [LDL-C T1 218.22±50.15, T2 163.85±27.64, T3 153.12±34.90, T4 156.37±34.11 mg/dl, p<0.05]. The mean reduction of LDL-C in comparison to baseline levels was: -34.68±12.99% at T2 and -30.42±20.78% at T4. Despite this efficacy and excluding one case of statin-intolerance, 9/23 patients (about 39%) dropped out after one year of statin therapy with a higher prevalence among families without an history of precocious cardio-vascular events (p <0.05). Conclusions. We report an overall scarce adherence to statins in our paediatric HeFH population despite an efficacy in line with international data. GP involvement, a more effective communication with patients and their families to emphasize the high HeFH-related cardiovascular risk, and a periodic follow-up including telemedicine may be tools to achieve a better adherence.