Key result
Lomitapide effectively lowers triglycerides and may benefit refractory genetic hypertriglyceridemia with recurrent acute pancreatitis.
Why the study?
Severe genetic forms of hypertriglyceridemia carry a risk of life-threatening pancreatitis and lack available effective treatments.
Does lomitapide reduce plasma triglycerides in patients with severe genetic forms of hypertriglyceridemia?
Does lomitapide reduce plasma triglycerides in patients with severe genetic forms of hypertriglyceridemia?
Lomitapide may be an effective treatment for lowering triglycerides in patients with severe genetic hypertriglyceridemia refractory to traditional therapies, though long-term hepatic safety remains a concern.
May warrant consideration in refractory genetic hypertriglyceridemia with pancreatitis; leaves open long-term hepatic safety and needs prospective trials.
INTRODUCTION: Severe genetic forms of hypertriglyceridemia carry a risk of life-threatening pancreatitis and lack available effective treatments. Lomitapide is a microsomal triglyceride transfer protein inhibitor currently approved for treatment of homozygous familial hypercholesterolemia that may be useful in the management of severe hypertriglyceridemia. Areas covered: Published trials of lomitapide that reported plasma triglyceride response were reviewed, as was a case report of a patient with hypertriglyceridemia who was treated for 13 years with lomitapide. ClinicalTrials.gov was also reviewed for any unpublished results and ongoing trials. Expert opinion: Lomitapide demonstrates effective triglyceride lowering and may be a useful treatment for patients with genetic hypertriglyceridemia and recurrent acute pancreatitis who are refractory to traditional treatment. However, long term hepatic safety may be a concern and direct clinical trial-level data are lacking for this indication.
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Brahm et al. (2016) conducted a review in Severe genetic forms of hypertriglyceridemia. Lomitapide was evaluated on Plasma triglyceride response. Lomitapide demonstrates effective triglyceride lowering and may be a useful treatment for patients with genetic hypertriglyceridemia and recurrent acute pancreatitis refractory to traditional treatment.
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