Effect of metreleptin treatment in congenital generalized lipodystrophy: a retrospective analysis from the MENA region.

ABSTRACT:

Purpose

Congenital generalized lipodystrophy (CGL) is characterized by near-total loss of body fat, leptin deficiency, and severe metabolic complications. Clinical data show that metreleptin improves metabolic control in CGL; however, data from the Middle East and North Africa (MENA) is limited. We assessed the real-world effectiveness and safety of metreleptin in patients with CGL from this region.

Methods

We conducted a multicenter, retrospective chart review of patients with CGL. Baseline, 6 ± 2months (short-term), 2 ± 1years (long-term), and >3 years follow-up data were analyzed. Median changes from baseline were calculated for short- and long-term assessments; >3 years data were reviewed individually. Safety was evaluated up to 90 days after last dose.

Results

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SUBMITTER: Alfadhel M 

PROVIDER: S-EPMC13627170 | biostudies-literature | 2026 Oct

REPOSITORIES: biostudies-literature