Session: SUN 839-872-Diabetes & Obesity Management
Poster Board SUN-850
Methods: We studied 5 patients with homozygous or compound heterozygous mutations of the alpha subunit of the insulin receptor, and a phenotype consistent with RMS, including extreme insulin resistance, dental anomalies, and short stature. In the 2 patients treated for 10 years, metreleptin was given initially at 0.02 mg/kg/day, and titrated up over years. These 2 patients underwent 3 cycles of leptin withdrawal and reinitiation, with the most recent cycle of metreleptin started at 0.22 mg/kg/day. In the other 3 patients, metreleptin was initiated at 0.22 mg/kg/day. Statistical analysis of metreleptin effects on A1c and body weight were tested using a 12 month period during which all patients were treated with 0.22 mg/kg/day and no changes were made in other diabetes medications.
Results: All patients had poorly controlled diabetes (A1c 10.1 to 12.8%) at metreleptin initiation. At baseline, A1c (mean±SD) was 11.4±1.1, and decreased to 8.9±2.0 at 5 months, 10.0±0.5 at 8 months, and 9.7±1.6 at 12 months after 0.22 mg/kg/day metreleptin (P=0.0035 for main effect of metreleptin on repeated measures ANOVA). Patients lost weight early in metreleptin treatment (-3.4 kg at 5 months, P=0.04), but regained this weight over time (-1.3 kg at 12 months compared to baseline, P=0.3). In the 2 patients treated for 10 years, each cycle of leptin initiation was associated with a decrease in A1c, and each withdrawal was associated in a rise in A1c.
Conclusions: Metreleptin treatment was associated with a decrease in A1c in 5 patients with RMS, and may be a promising treatment option for this difficult to control disease.
Nothing to Disclose: RJB, EC, PG
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