In Reply We appreciate the opportunity to comment on the points raised by Miyares. First, we emphasize that use of lomitapide for any type of hyperlipidemia requires careful monitoring for hepatoxicity. In the case of familial chylomicronemia that we reported,1 changes in enzymes and biopsy specimens associated with hepatoxicity began to occur after about 9 years of use and became moderate to severe after 13 years. No other approved effective treatments could have been used, and lomitapide was made available under compassionate use. Currently, the patient is being followed up closely by a hepatologist and her clinical team.
Sacks FM, Stanesa M, Hegele RA. Progression to Hepatitis and Fibrosis Secondary to Lomitapide Use—Reply. JAMA Intern Med. 2014;174(9):1522-1523. doi:10.1001/jamainternmed.2014.1528