Summary

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• Myalept (metreleptin) is used as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy.
• This summary is based on the review of two systematic review(s)/meta-analysis(es). ^[1-2]
• Metreleptin reduces triglycerides and HbA1c in all lipodystrophy cases (n = 111) and in LMNA, PPARG, AGPAT2, and BSCL2 subgroups (n = 72, 13, 21, and 21, respectively). It lowers BMI in partial/generalized lipodystrophy and in LMNA or BSCL2 subgroups but not in PPARG or AGPAT2 subgroups.
• Thiazolidinediones improve HbA1c and triglycerides in all lipodystrophy cases (n = 13) and show specific effectiveness in PPARG for HbA1c (n = 5) and LMNA for triglycerides (n = 7). Recombinant Human Insulin-like Growth Factor-1 (rhIGF-1) improves HbA1c in INSR-related insulin resistance (n = 17).
• Eleven LEP deficiency patients benefitted from long-term metreleptin treatment, with one patient developing neutralizing antibodies leading to a loss of efficacy.
• Safety outcomes beyond the development of neutralizing antibodies and hyperinsulinemia in LEP deficiency patients were not explicitly detailed in the reviewed studies.
• Metreleptin shows effectiveness in LMNA, PPARG, AGPAT2, and BSCL2 genetic subgroups and benefits patients with LEP deficiency, though there is a risk of developing neutralizing antibodies leading to loss of efficacy; LEP deficiency patients are diagnosed younger with higher BMI SD scores and higher rates of hyperinsulinemia compared to LEPR deficiency patients, who are associated with missense variants.