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.
Domains
Endocrinology and metabolism
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