Rare Antagonistic Leptin Variants and Severe, Early-Onset Obesity
Study context
- Population
- Two unrelated children with homozygous antagonistic leptin variants, intense hyperphagia, severe obesity and high circulating leptin
- Participants
- 2 unrelated children
- Outcome
- Characterization of leptin variants and response to recombinant leptin
Main finding
As reported in the abstract
- Both variants bind to the leptin receptor but trigger marginal, if any, signaling
- In presence of nonvariant leptin, the variants act as competitive antagonists
- Recombinant leptin started at high doses, gradually lowered; both patients eventually attained near-normal weight
Safety
Safety of recombinant leptin
- Severe adverse events
- 0
- Antidrug antibodies developed, with no apparent effect on efficacy
Original publication
Rare Antagonistic Leptin Variants and Severe, Early-Onset Obesity. N Engl J Med. Published 15 June 2023. doi:10.
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