| Abstract | CONTEXT: Acquired generalized lipodystrophy (AGL) is marked by severe insulin resistance and hypertriglyceridemia. Rarely, AGL and type 1 diabetes (T1D) coexist. OBJECTIVE: Our objective was to describe the response to leptin therapy in patients with coexisting AGL and T1D and to document the autoimmune diseases associated with AGL. DESIGN AND SETTING: We conducted an open-label prospective study at the Clinical Research Center of the National Institutes of Health. PATIENTS: Participants included 50 patients with generalized or partial lipodystrophy (acquired or congenital); two patients had both AGL and T1D. INTERVENTION: Patients were treated with 12 months of recombinant human leptin administration to achieve high-normal serum concentrations. RESULTS: Two patients had both AGL and T1D. The first was diagnosed with T1D at age 8 yr. Beginning at age 11 yr, he developed generalized lipodystrophy, elevated transaminases, and poor glycemic control [hemoglobin A 1c (HbA 1c) 10.7%] despite markedly increased insulin requirements (3.3-5 U/kg.d). Further evaluation revealed hypoleptinemia and hypertriglyceridemia. At age 15 yr, leptin therapy was initiated, and after 1 yr, his insulin requirements fell to 1 U/kg.d, his glycemic control improved (HbA 1c 8.4%), and both his triglycerides and transaminases normalized. The second patient developed concurrent AGL and T1D at age 6 yr. Despite insulin doses of up to 32 U/kg.d, she developed poor glycemic control (HbA 1c 10.6%), hypertriglyceridemia (2984 mg/dl), elevated transaminases, and nonalcoholic steatohepatitis. At age 13 yr, leptin therapy was started, and after 1 yr, her glycemic control improved (HbA 1c 7.3%) and her insulin requirements decreased (17 U/kg.d). Her triglycerides remained elevated but were improved (441 mg/dl). CONCLUSIONS: Long-term recombinant leptin therapy is effective in treating the insulin resistance of patients with the unusual combination of T1D and AGL. |
| Authors | Jean Y Park, Angeline Y Chong, Elaine K Cochran, David E Kleiner, Michael J Haller, Desmond A Schatz, Phillip Gorden
(Affiliation: Clinical Endocrinology Branch, National Institute of Diabetes and Digestive and Kidney Diseases, National Cancer Institute, National Institutes of Health, Bethesda, Maryland 20892, USA.)
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| Journal | The Journal of clinical endocrinology and metabolism
(J Clin Endocrinol Metab)
Vol. 93
Issue 1
Pg. 26-31
(Jan 2008)
ISSN: 0021-972X United States |
| PMID | 17940115
(Publication Type: Case Reports, Journal Article, Research Support, N.I.H., Intramural)
|
| Chemical References |
- Blood Glucose
- Hemoglobin A, Glycosylated
- Leptin
- Recombinant Proteins
- Triglycerides
- Aspartate Aminotransferases
- Alanine Transaminase
|
| Topics |
- Adolescent
- Alanine Transaminase
(blood)
- Aspartate Aminotransferases
(blood)
- Blood Glucose
(metabolism)
- Diabetes Mellitus, Type 1
(blood, complications, drug therapy)
- Fatal Outcome
- Female
- Hemoglobin A, Glycosylated
(metabolism)
- Humans
- Hypertriglyceridemia
(blood, complications, drug therapy)
- Insulin Resistance
(physiology)
- Leptin
(blood, therapeutic use)
- Lipodystrophy
(blood, complications, drug therapy)
- Male
- Prospective Studies
- Recombinant Proteins
(blood, therapeutic use)
- Triglycerides
(blood)
|
