Highlights
- •We describe the first use of lomitapide in a HoFH child for 4 years.
- •20 mg/d lomitapide added extra 37% reduction in LDL-C.
- •Lomitapide was well tolerated.
- •Despite treatment carotid and aortic valve disease progressed.
Abstract
We report for the first time the efficiency and safety of a 49-month compassionate
use of the microsomal transfer protein inhibitor lomitapide in a child with homozygous
familial hypercholesterolemia. On average, 20 mg of lomitapide caused a 37% reduction
in low-density lipoprotein cholesterol levels on top of ezetimibe and atorvastatin.
The drug was well tolerated with no changes in liver enzymes and occurrence of steatosis
on hepatic ultrasound. The patient presented adequate growth and sexual maturation.
Nonetheless, there was progression in either subclinical atherosclerotic carotid or
aortic valve diseases. Further studies are necessary to test the impact and safety
of lomitapide in children with homozygous familial hypercholesterolemia.
Keywords
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Article info
Publication history
Published online: March 11, 2019
Accepted:
March 3,
2019
Received:
October 22,
2018
Identification
Copyright
© 2019 National Lipid Association. All rights reserved.
