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Ned Tijdschr Geneeskd · Jan 2013
Review Case Reports[Therapy resistant diabetes mellitus and lipodystrophy: leptin therapy leads to improvement].
- Ingrid M Jazet, Jacqueline T Jonker, Marjolein A Wijngaarden, Hildo Lamb, and August H M Smelt.
- Leids Universitair Medisch Centrum, Afd. Endocrinologie/Algemene Interne Geneeskunde, Leiden, the Netherlands. i.m.jazet@lumc.nl
- Ned Tijdschr Geneeskd. 2013 Jan 1; 157 (4): A5482.
AbstractLipodystrophy is a congenital or acquired disorder characterized by complete or partial absence of subcutaneous fat tissue, often accompanied by insulin resistance, diabetes mellitus (DM), hypertriglyceridemia and hepatic steatosis. A decrease in both number and function of adipocytes leads to ectopic fat depositions and decreased production of adipokines such as leptin. We present 2 patients with inadequately regulated DM, hypertriglyceridemia and hepatic steatosis who were eventually diagnosed with lipodystrophy: 1 with congenital generalized lipodystrophy (Berardinelli-Seip syndrome) and 1 with congenital partial lipodystrophy (Dunnigan syndrome). Both received recombinant human leptin therapy (methionylleptin, available on a compassionate-use basis). This resulted in improved plasma levels of triglyceride, glucose and HbA1c and a decrease in liver size. In addition, hepatic triglyceride content decreased from 19.3% to 1.3% in the first patient and from 20.6% to 12.4% in the second. Leptin therapy is an effective and safe treatment for therapy-resistant diabetes and hypertriglyceridemia in patients with congenital lipodystrophy.
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