The Endocrine Society

The Endocrine Society

April 12, 2017
1 min read

Myalept reduced liver fat content in patients with partial lipodystrophy

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ORLANDO, Fla. Patients with partial lipodystrophy-associated non-alcoholic steatohepatitis responded positively to metreleptin therapy, according to a study presented at ENDO 2017.

“Fatty liver, or excess fat building up in the liver, is a common metabolic disturbance seen in patients with lipodystrophy,” Elif A. Oral, MD, associate professor of endocrinology at Michigan Medicine said in a press release. “The underlying metabolic disturbances seen in this patient population can be difficult to manage with traditional therapies.”

Oral and colleagues enrolled 23 patients in the study to test the efficacy of Myalept (metreleptin, Aegerion Pharmaceuticals) in treating partial lipodystrophy. Twenty-two patients were diagnosed with NASH following biopsy, 19 patients completed the 12 months of therapy and 18 completed the secondary biopsy.

Mean NASH score at baseline was 6 and was reduced to 5 at 12 months (P = .0079). Mean non-alcoholic fatty liver disease activity score (NAS) at baseline was 5 and was reduced to 4 at 12 months (P = .0002). Mean liver fat quantification at baseline was 13.3% and was reduced to 8.4% at 12 months (P = .0014).

Nine of the final 18 patients responded to the therapy with a mean liver fat reduction of 49% (P = .0047). The responders had a lower baseline leptin level (14.5 ng/mL) compared with the non-responders (25 ng/mL; P = .0539).

Adverse events that were considered possibly or probably related to the therapy included 11 mild events, nine moderate events and two severe events (hypoglycemia and nausea/vomiting).

The researchers note that the low number and heterogeneous nature of the population, as well as the patients’ complicated medical history, were considerable limitations in the study.

“About half of the patients had scores that lowered by two points or more, which is clinically significant in patients with this disease,” Oral added in the release. “Generally, that type of drop is only seen with 10% or more sustained weight loss in the common form of fatty liver disease, which usually only occurs with metabolic surgery.” – by Talitha Bennett


Ajluni N, et al. Abstract OR09-4. Presented at: ENDO 2017; Apr. 1-4, 2017; Orlando.

Disclosure: Oral reports she received grant and drug support from and served as an advisor for Amylin Pharmaceuticals, Bristol-Myers Squibb, AstraZeneca, Aegerion Pharmaceuticals, Akcea Therapeutics and Ionis Pharmaceuticals. The remaining researchers report no relevant financial disclosures.