Dermatologic Manifestations of Generalized Lipodystrophy Treatment & Management

Updated: Jun 17, 2022
  • Author: Camila K Janniger, MD; Chief Editor: Dirk M Elston, MD  more...
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Medical Care

Successful treatment of acanthosis nigricans (AN) in association with generalized lipodystrophy was accomplished in 1 patient with etretinate beginning at 75 mg/d. [21] Therapy for AN was undertaken in this patient for cosmetic reasons. After 4 weeks, the cutaneous eruption had disappeared, with only modest elevation of the preexisting hyperlipemia.

Substitution of eucaloric medium-chain triglycerides for long-chain fatty acids in the diet of 1 patient led to improvement of chylomicronemia, xanthomatosis, hypertriglyceridemia, hepatomegaly, carbohydrate tolerance, and hyperinsulinemia, but not lipoatrophy. Dietary fish oil also may be useful in improving AN.

Because conventional lipid-lowering and antihyperglycemic medications may be insufficient to control severe metabolic abnormalities, leptin has been used for severe lipodystrophy and found to significantly improve metabolic abnormalities. [22] This new option for therapy is leptin, an adipocyte hormone, which may improve insulin resistance, hyperglycemia, dyslipidemia, and hepatic steatosis. [23] Leptin is the first of a group of adipocyte-secreted hormones to be used clinically. [24] Its long-term efficacy in 15 patients with generalized lipodystrophy was evaluated. [25]

Leptin has been judged the first novel, effective, long-term treatment for severe forms of lipodystrophy. Treating severe lipodystrophy as a leptin deficiency syndrome has been shown to improve the metabolic outcomes in those affected patients. [26]

In February 2014, the FDA approved metreleptin (Myalept), a recombinant leptin analog, for congenital or acquired generalized lipodystrophy in children and adults. [27] The approval was based on results from an NIH open-label, single-arm study that included 48 patients with congenital or acquired generalized lipodystrophy who also had diabetes mellitus, hypertriglyceridemia, and/or elevated levels of fasting insulin. The trial showed reductions in fasting glucose and triglycerides. [17]

Metreleptin is not indicated for the following:

  • Treatment of complications of partial lipodystrophy

  • Treatment of liver disease, including nonalcoholic steatohepatitis (NASH)

  • HIV-related lipodystrophy

  • Use in patients with metabolic disease, including diabetes mellitus and hypertriglyceridemia, without concurrent evidence of generalized lipodystrophy