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Therapeutic indications and metabolic effects of metreleptin in patients with lipodystrophy syndromes: Real-life experience from a national reference network

Abstract : Aims: To describe baseline characteristics and follow-up data in patients with lipodystrophy syndromes treated with metreleptin in a national reference network, in a real-life setting. Patients and Methods: Clinical and metabolic data from patients receiving metreleptin in France were retrospectively collected, at baseline, one year and at the latest follow-up during treatment. Results: Forty-seven patients with lipodystrophy including generalized lipodystrophy (GLD, n=28) and partial lipodystrophy (PLD, n=19) received metreleptin over the last decade. At baseline, age was 29.3 [16.6-47.6] (years, median [interquartile range]); BMI 23.8 kg/m2 [21.2-25.7]; serum leptin 3.2 [1.0- 4.9] ng/mL); 94% of patients had diabetes (66% insulin-treated), 53% hypertension, and 87% dyslipidemia. Metreleptin therapy, administered during 31.7 [14.2-76.0] months, was ongoing in 77% of patients at the latest follow-up. In patients with GLD, HbA1c (%) and fasting triglycerides (mmol/L) significantly decreased from baseline to one year-metreleptin treatment, from 8.4 [6.5-9.9] to 6.8 [5.6- 7.4], and 3.6 [1.7-8.5] to 2.2 [1.1-3.7] respectively (p<0.001), with a sustained efficacy thereafter. In patients with PLD, HbA1c (%) was not significantly modified from 7.7 [7.1-9.1] at baseline vs 7.7 [7.4- 9.5] at one-year), and the decrease in fasting triglycerides (mmol/L), from 3.3 [1.9-9.9] to 2.5 [1.6-5.3], p<0.01) was not confirmed at the latest assessment (5.2 [2.2-11.3]). However, among PLD patients, at one-year, 61 % were responders regarding glucose homeostasis, with lower baseline leptin levels compared to non-responders, and 61% were responders regarding triglyceridemia. Liver enzymes significantly decreased only in the GLD group. Conclusions: In this real-life setting study, metabolic outcomes are improved under metreleptin therapy in patients with GLD. The therapeutic indication of metreleptin needs to be clarified in patients with PLD.
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https://hal.sorbonne-universite.fr/hal-03649731
Contributor : Corinne Vigouroux Connect in order to contact the contributor
Submitted on : Friday, April 22, 2022 - 6:53:22 PM
Last modification on : Thursday, August 4, 2022 - 5:22:46 PM
Long-term archiving on: : Saturday, July 23, 2022 - 7:27:43 PM

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H Mosbah, M Vantyghem, E Nobécourt, F Andreelli, F Archambeaud, et al.. Therapeutic indications and metabolic effects of metreleptin in patients with lipodystrophy syndromes: Real-life experience from a national reference network. Diabetes, Obesity and Metabolism, Wiley, 2022, ⟨10.1111/dom.14726⟩. ⟨hal-03649731⟩

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