Physicians' Academy for Cardiovascular Education

Statin treatment effective in pediatric patients with homozygous FH

Efficacy of Rosuvastatin in Children With Homozygous Familial Hypercholesterolemia and Association With Underlying Genetic Mutations

Stein EA, Dann EJ, Wiegman A, et al. - J Am Coll Cardiol. 2017;70(9):1162-1170

Background

Rosuvastatin reduces significantly LDL-C levels in adults with homozygous familial hypercholesterolemia (HoFH), but data in pediatric patients are limited [1]. There is also a lack of data regarding the use of lomitapide, mipomersen and evolocumab, as well as apheresis in young HoFH children [2,3].

In this global study, the efficacy and safety of rosuvastatin on LDL-C and other lipids, lipoproteins, and apolipoproteins was evaluated compared with placebo, in 13 children and adolescents aged < 18 years with HoFH. Moreover, the association between HoFH genotypes and the LDL-C response to rosuvastatin was assessed in a broader HoFH population of both children and adults.

Patients with at least 1 of the following were eligible for the study:

- tendon or cutaneous xanthoma before the age of 10 years

- HeFH diagnosed by genetic or clinical criteria in both parents

Eligible patients discontinued all lipid-lowering therapy except ezetimibe and/or apheresis, and received rosuvastatin 10 mg daily for 4 weeks in the lead-in phase. Subsequently, they were randomized 1:1 to a double-blind, 12-week crossover period of rosuvastatin 20 mg daily versus placebo, followed by a 12-week, open-label rosuvastatin 20 mg maintenance phase.

Main results

Conclusion

In children and adolescents with HoFH, rosuvastatin 20 mg alone or in combination with ezetimibe and/or apheresis led to an effective LDL-C reduction, and was well tolerated. Consequently, rosuvastatin is approved for the treatment of HoFH pediatric patients 7 to 17 years of age. The LDL-C response to rosuvastatin was related to the underlying mutations and was consistent with that seen in adults.

References

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