Impact of Early Evidence of Atherosclerotic Changes on Early Treatment in Children With Familial Hypercholesterolemia

Prevention of cardiovascular disease in patients with familial hypercholesterolaemia: The role of PCSK9 inhibitors

“… 57 Data suggest, however, that it may be prudent to initiate cholesterol screening in children, in whom elevated levels of LDL-cholesterol alone are strongly diagnostic of FH. 59 In addition, screening of children has been shown to be an effective method for the diagnosis of affected parents and siblings through a cascade approach. 60 Furthermore, identifying FH in childhood enables treatment to be initiated early, which could result in improved long-term outcomes for patients, although research is needed to ascertain the exact age to begin treatment and the long-term safety of LLTs.…”

Management of patients with familial hypercholesterolaemia

“…Despite these potential concerns, no published reports indicate that statin treatment of children with FH causes harm owing to inhibition of endogenous cholesterol synthesis. 108 Results from an international, 2-year, open-label, titration-togoal study, which will include 198 children with HeFH aged 6-18 years, should clarify these concerns. 109 In contrast to HeFH, children with HoFH should be treated as early as possible (no lower limit of age) because they have extremely high risk of premature CVD and death.…”

Clinical guidance on the contemporary use of proprotein convertase subtilisin/kexin type 9 monoclonal antibodies

“…For children and adolescents, who would generally require PCSK9 therapy due to FH, current recommendations are for statin treatment from 8 years of age, although a previous report describes a homozygous FH patient being treated from 3 years of age with no long‐term complications reported at 11 years . The use of PCSK9 inhibitors in children and adolescents remains uncertain and is not currently recommended .…”