Children with familial hypercholesterolemia do not reach treatment targets

Gisle Langslet‘s PhD thesis shows that statins are safe and effective, but underused in children and young adults with familial hypercholesterolemia.

MAIN RESULTS:

Cholesterol-lowering statins are safe and effective for children who have inherited high cholesterol.
Statins reverses the increased carotid intima media thickness in children with familial hypercholesterolemia.
Several children and young adults with familial hypercholesterolemia are not adequately treated.

THESIS DEFENCE:

Thesis: Treatment and long term follow-up of children and young adults with familial hypercholesterolemia
Candidate: Gisle Langslet
Time: April 24, 2018 at 12:30
Place: Oslo University Hospital, Rikshospitalet B: Red auditorium
Link to university website (in Norwegian)

SUMMARY:

(1/4) Two years treatment with rosuvastatin lowered LDL cholesterol with up to 45 % in children and adolescents with familial hypercholesterolemia. Statin treatment proved to be safe and did not influence neither growth nor puberty.

The study includes 197 children between 6 and 16 years old from The Netherlands, Norway, Canada, The US and Belgium. These children had higher baseline carotid intima media thickness than their siblings without familial hypercholesterolemia, but two years of rosuvastatin treatment eliminated this difference.

(3) Also atorvastatin is safe and effective for children with familial hypercholesterolemia. This study was done in collaboration with American and Greek researchers, and inclued 272 6–15-year-olds with familial hypercholesterolemia.

After three years of treatment, LDL cholesterol was lowered by a mean of 44 % in the youngest children and 40 % in the eldest. The treatment did not affect growth or maturation, and only 2 % of the children quit treatment due to side effects.

(2) Several young adults with familial hypercholesterolemia discontinue statin treatment. 67 adult Norwegians were followed up approximately ten years after participation in a clinical study, at which point 28 % of the participants did not use statins anymore.

The ones who did not use cholesterol-lowering medication had higher levels of LDL cholesterol than those who were still on statins. In total however, only 9 % of the participants had reached their target cholesterol level of 2.5 mmol/L. The levels were lower in patients who had been followed up during the past two years.

(5) Just a little more than half of Norwegian children with familial hypercholesterolemi use cholesterol-lowering medication. 302 children were included, and less than half of them had reached the treatment target at last follow-up. Among those who did not use statins, only 22 % had recommended levels of LDL cholesterol.

REFERENCES:

(1) Braamskamp, M. J., Langslet, G., McCrindle, B. W., Cassiman, D., Francis, G. A., Gagné, C., Gaudet, D., Morrison, K. M., Wiegman, A., Turner, T., Kusters, D. M., Miller, E., Raichlen, J. S., Wissmar, J., Martin, P. D., Stein, E. A., & Kastelein, J. J. P., (2015). Efficacy and safety of rosuvastatin therapy in children and adolescents with familial hypercholesterolemia: Results from the CHARON study. Journal of clinical lipidology, 9(6), 741-750.

(2) Langslet, G., Bogsrud, M. P., Halvorsen, I., Fjeldstad, H., Retterstøl, K., Veierød, M. B., & Ose, L. (2015). Long-term follow-up of young adults with familial hypercholesterolemia after participation in clinical trials during childhood. Journal of clinical lipidology, 9(6), 778-785.

(3) Langslet, G., Breazna, A., & Drogari, E. (2016). A 3-year study of atorvastatin in children and adolescents with heterozygous familial hypercholesterolemia. Journal of clinical lipidology, 10(5), 1153-1162.

(4) Braamskamp, M. J., Langslet, G., McCrindle, B. W., Cassiman, D., Francis, G. A., Gagne, C., Gaudet, D., Morrison, K. M., Wiegman, A., Turner, T., Miller, E., Kusters, D. M., Raichlen, J. S., Martin, P. D., Stein, E. A., Kastelein, J. J. P., & Hutten, B. A. (2017). Effect of Rosuvastatin on Carotid Intima-Media Thickness in Children With Heterozygous Familial HypercholesterolemiaClinical Perspective: The CHARON Study (Hypercholesterolemia in Children and Adolescents Taking Rosuvastatin Open Label). Circulation, 136(4), 359-366.

(5) Bogsrud, M. P., Langslet, G., Wium, C., Johansen, D., Svilaas, A., & Holven, K. B. (2017). Treatment goal attainment in children with familial hypercholesterolemia: A cohort study of 302 children in Norway. Journal of clinical lipidology.