What is the incidence of statin-associated symptoms and statin intolerance in children with familial hypercholesterolemia?
Statin therapy is generally well-tolerated in children with familial hypercholesterolemia, with true statin intolerance being rare (1.9%) despite frequent but mild and transient statin-associated symptoms.
AbstractBackground and aims To reduce the increased premature cardiovascular risk in children with heterozygous familial hypercholesterolemia (FH), statins are the first pharmacological step. This study aims to assess the incidence of statin-associated symptoms (SAS) and statin intolerance in pediatric FH patients. Methods Children (Results A total of 696 children were included (329 girls, 47.3%) with a median age (IQR) at statin initiation of 11.0 (8.3 – 14.0) years. SAS were reported in 1 in 8.5 children at T1, 1 in 8.2 children at T2, and 1 in 8.2 at T3. The majority of symptoms were transient and mild. Muscle symptoms without clinically significant creatine kinase (CK) elevation (≤3× upper limit of normal) were most frequently reported. No cases of rhabdomyolysis or other adverse events requiring hospital admission were reported. In total, 13 children (1.9%) were statin intolerant due to persistent symptoms leading to permanent discontinuation of statin therapy. Conclusions This study shows that SAS were common, but generally transient and mild, whereas statin intolerance was relatively uncommon in children with FH. Proactive management of SAS and consideration of alternative therapies are crucial to maintain adherence and reduce their increased cardiovascular risk. Future research should focus on identifying risk factors to enable personalized treatment strategies.
Bosch et al. (Sun,) studied this question.