[Therapy resistant diabetes mellitus and lipodystrophy: leptin therapy leads to improvement].
Lipodystrophy 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.
Leids Universitair Medisch Centrum, Afd. Endocrinologie/Algemene Interne Geneeskunde, Leiden, the Netherlands. i.m.jazet@lumc.nl
Ingrid M
IM
Jazet
Jacqueline T
JT
Jonker
Marjolein A
MA
Wijngaarden
Hildo
H
Lamb
August H M
AH
Smelt
D000293:Adolescent; D000328:Adult; D001786:Blood Glucose / Q000378:metabolism; D057176:Compassionate Use Trials; D003920:Diabetes Mellitus / Q000188:drug therapy / Q000378:metabolism; D005234:Fatty Liver / Q000150:complications / Q000188:drug therapy / Q000378:metabolism; D005260:Female; D006442:Glycated Hemoglobin / Q000032:analysis; D006801:Humans; D015228:Hypertriglyceridemia / Q000150:complications / Q000188:drug therapy / Q000378:metabolism; D007333:Insulin Resistance; D020738:Leptin / Q000172:deficiency / Q000627:therapeutic use*; D008060:Lipodystrophy / Q000150:complications / Q000175:diagnosis / Q000188:drug therapy* / Q000378:metabolism; D052497:Lipodystrophy, Congenital Generalized / Q000150:complications / Q000175:diagnosis / Q000188:drug therapy* / Q000378:metabolism; D014280:Triglycerides / Q000097:blood
D001786:Blood Glucose; D006442:Glycated Hemoglobin; D020738:Leptin; D014280:Triglycerides