Statins for children with familial hypercholesterolemia

Abstract

Statin treatment is an efficient lipid-lowering therapy in children with familial hypercholesterolemia. No significant safety issues were identified. Statin treatment seems to be safe in the short term, but long-term safety is unknown. Children treated with statins should be carefully monitored and followed up by their pediatricians or physicians into adulthood. Large long-term randomized controlled trials are needed to establish the long-term safety issues of statins.

A 3-year study of atorvastatin in children and adolescents with heterozygous familial hypercholesterolemia

“…The reductions of LDL-C observed are in accordance with the findings from other clinical studies conducted in pediatric and adolescent populations in which atorvastatin and other statins have been administered. 17,[19][20][21][22][23][24][25][26]28 Moreover, the early efficacy of atorvastatin in this study is consistent with that observed in a similar but smaller (n 5 39) short-term (8-week) atorvastatin study in which doses of 5-20 mg per day were administered to children and adolescents with HeFH. In the small 8-week study, mean LDL-C reductions from baseline of 40.7% and 39.7% were observed at week 8 in subjects at TS 1 and $2, respectively.…”

A 3-year study of atorvastatin in children and adolescents with heterozygous familial hypercholesterolemia

“…The reductions of LDL-C observed are in accordance with the findings from other clinical studies conducted in pediatric and adolescent populations in which atorvastatin and other statins have been administered. 17,[19][20][21][22][23][24][25][26]28 Moreover, the early efficacy of atorvastatin in this study is consistent with that observed in a similar but smaller (n 5 39) short-term (8-week) atorvastatin study in which doses of 5-20 mg per day were administered to children and adolescents with HeFH. In the small 8-week study, mean LDL-C reductions from baseline of 40.7% and 39.7% were observed at week 8 in subjects at TS 1 and $2, respectively.…”

Effectiveness and Safety of Statin Therapy in Children: A Real-World Clinical Practice Experience

“…Despite continuous follow‐up and self‐reported high compliance rates, minimal (3.4 mmol/L) and optimal (2.9 mmol/L) LDL‐C treatment goals as defined by pediatric guidelines were reported in only 49% and 14% of patients, respectively 1–3 , 26 . These results are similar to those reported in most RCTs and clinical case series, 15–17 , 19 , 20 , 22–33 with the exception of a single pediatric preventive cardiology program that targeted these thresholds for more‐intensive patient management 21 . Of note, the average LDL‐C decrease of 36% in this series was similar to the 32% reported in the RCT cohort follow‐up of Luirink et al 9 .…”

“…45 In meta-analyses of statin trials in children, incidence of hepatic enzyme elevation did not differ between statin-treated and placebo groups, and there was no diagnosis of liver disease. 14,15 In our series, serial evaluation of hepatic enzymes showed no evidence of sustained elevation. Reported findings of muscle toxicity in adults range from asymptomatic increases in creatinine kinase levels, to myalgia without CK elevation, to myositis with CK elevation; extremely rarely, statin treatment has been associated with rhabdomyolysis.…”

Screening for Familial Hypercholesterolemia in Children: What Can We Learn From Adult Screening Programs?

“…With the pathological changes of FH emerging in early childhood, FH may be considered an exception. Interventions could begin earlier to promote a healthy lifestyle and statin treatment from as early as age 8-10 years [17,30]. This is consistent with the recent recommendation of the American Academy of Pediatrics that "Predictive genetic testing for adult onset conditions generally should be deferred unless an intervention initiated in childhood may reduce morbidity or mortality" [48].…”

A 3-year study of atorvastatin in children and adolescents with heterozygous familial hypercholesterolemia

“…The reductions of LDL-C observed are in accordance with the findings from other clinical studies conducted in pediatric and adolescent populations in which atorvastatin and other statins have been administered. 17,[19][20][21][22][23][24][25][26]28 Moreover, the early efficacy of atorvastatin in this study is consistent with that observed in a similar but smaller (n 5 39) short-term (8-week) atorvastatin study in which doses of 5-20 mg per day were administered to children and adolescents with HeFH. In the small 8-week study, mean LDL-C reductions from baseline of 40.7% and 39.7% were observed at week 8 in subjects at TS 1 and $2, respectively.…”

Effectiveness and Safety of Statin Therapy in Children: A Real-World Clinical Practice Experience

“…Despite continuous follow‐up and self‐reported high compliance rates, minimal (3.4 mmol/L) and optimal (2.9 mmol/L) LDL‐C treatment goals as defined by pediatric guidelines were reported in only 49% and 14% of patients, respectively 1–3 , 26 . These results are similar to those reported in most RCTs and clinical case series, 15–17 , 19 , 20 , 22–33 with the exception of a single pediatric preventive cardiology program that targeted these thresholds for more‐intensive patient management 21 . Of note, the average LDL‐C decrease of 36% in this series was similar to the 32% reported in the RCT cohort follow‐up of Luirink et al 9 .…”

“…45 In meta-analyses of statin trials in children, incidence of hepatic enzyme elevation did not differ between statin-treated and placebo groups, and there was no diagnosis of liver disease. 14,15 In our series, serial evaluation of hepatic enzymes showed no evidence of sustained elevation. Reported findings of muscle toxicity in adults range from asymptomatic increases in creatinine kinase levels, to myalgia without CK elevation, to myositis with CK elevation; extremely rarely, statin treatment has been associated with rhabdomyolysis.…”

Screening for Familial Hypercholesterolemia in Children: What Can We Learn From Adult Screening Programs?

“…With the pathological changes of FH emerging in early childhood, FH may be considered an exception. Interventions could begin earlier to promote a healthy lifestyle and statin treatment from as early as age 8-10 years [17,30]. This is consistent with the recent recommendation of the American Academy of Pediatrics that "Predictive genetic testing for adult onset conditions generally should be deferred unless an intervention initiated in childhood may reduce morbidity or mortality" [48].…”

A 3-year study of atorvastatin in children and adolescents with heterozygous familial hypercholesterolemia

“…The reductions of LDL-C observed are in accordance with the findings from other clinical studies conducted in pediatric and adolescent populations in which atorvastatin and other statins have been administered. 17,[19][20][21][22][23][24][25][26]28 Moreover, the early efficacy of atorvastatin in this study is consistent with that observed in a similar but smaller (n 5 39) short-term (8-week) atorvastatin study in which doses of 5-20 mg per day were administered to children and adolescents with HeFH. In the small 8-week study, mean LDL-C reductions from baseline of 40.7% and 39.7% were observed at week 8 in subjects at TS 1 and $2, respectively.…”

Effectiveness and Safety of Statin Therapy in Children: A Real-World Clinical Practice Experience

“…Despite continuous follow‐up and self‐reported high compliance rates, minimal (3.4 mmol/L) and optimal (2.9 mmol/L) LDL‐C treatment goals as defined by pediatric guidelines were reported in only 49% and 14% of patients, respectively 1–3 , 26 . These results are similar to those reported in most RCTs and clinical case series, 15–17 , 19 , 20 , 22–33 with the exception of a single pediatric preventive cardiology program that targeted these thresholds for more‐intensive patient management 21 . Of note, the average LDL‐C decrease of 36% in this series was similar to the 32% reported in the RCT cohort follow‐up of Luirink et al 9 .…”

“…45 In meta-analyses of statin trials in children, incidence of hepatic enzyme elevation did not differ between statin-treated and placebo groups, and there was no diagnosis of liver disease. 14,15 In our series, serial evaluation of hepatic enzymes showed no evidence of sustained elevation. Reported findings of muscle toxicity in adults range from asymptomatic increases in creatinine kinase levels, to myalgia without CK elevation, to myositis with CK elevation; extremely rarely, statin treatment has been associated with rhabdomyolysis.…”

Screening for Familial Hypercholesterolemia in Children: What Can We Learn From Adult Screening Programs?

“…With the pathological changes of FH emerging in early childhood, FH may be considered an exception. Interventions could begin earlier to promote a healthy lifestyle and statin treatment from as early as age 8-10 years [17,30]. This is consistent with the recent recommendation of the American Academy of Pediatrics that "Predictive genetic testing for adult onset conditions generally should be deferred unless an intervention initiated in childhood may reduce morbidity or mortality" [48].…”