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Study · Observational study · n = 4 · 2025

8 results

Real-life experience on efficacy and safety of setmelanotide treatment in prepubertal children.

Kahveci A, Kurt I, Turan S, Guran T, Bereket A, Haliloglu B

European journal of endocrinology1 Feb 2025Cited by 1 (Europe PMC)

PMID39891402 (opens in a new tab)DOI10.1093/ejendo/lvaf008 (opens in a new tab)PepBankPUB-39891402
Publisher pagePubMedAsk about this study
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Summary

The text of this summary is not shown here, because the article license does not allow reuse. Read it at the source (opens in a new tab)

Text rights: Publisher copyright. Results still quote short attributed sentences.

Results from this study8

8 results from studies that gave the peptide. 8 results from people.

  1. 01HumanObservational studyEfficacy outcome

    In prepubertal children, effective setmelanotide doses were lower with POMC deficiency (0.3-0.5 mg/day) than LEPR deficiency (2.5 mg/day).

    In plain wordsIn prepubertal children, setmelanotide was effective at lower doses with POMC deficiency than with LEPR deficiency.

    Peptide
    Setmelanotide
    Outcome
    DecreaseEffective dose by genotype
    Study

    n: not reported in source

    Comparator
    LEPR deficiency
    Outcome group
    Overall treatment effect · Metabolic and weight
    Studied dose
    0.3-0.5 mg/day in POMC deficiency; 2.5 mg/day in LEPR deficiencyAs given in this study (human).

    Measurements

    Effective setmelanotide dose
    0.3 to 0.5 mg/day
    Patients with POMC deficiency
    Effective setmelanotide dose
    2.5 mg/day
    Patients with LEPR deficiency

    Source sentences

    Summary
    Findings indicate that setmelanotide is effective at lower doses in our patients with POMC deficiency (0.3-0.5 mg/day) than the patients with LEPR deficiency (2.5 mg/day).
    1 context sentence
    Summary · context
    This report presents real-life experiences on the efficacy and safety of setmelanotide, an MC4R agonist, in 4 prepubertal children (ages 3-9) with LEPR and POMC deficiencies.
  2. 02HumanCase report or seriesSafety outcome

    In prepubertal children with LEPR or POMC deficiency, setmelanotide was generally well tolerated.

    In plain wordsIn prepubertal children with LEPR or POMC deficiency, setmelanotide was generally well tolerated.

    Peptide
    Setmelanotide
    Outcome
    Not applicableTolerability
    Study

    n = 4

    Outcome group
    Adverse events · Whole body
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Treatment was generally well-tolerated, with injection site reactions and hyperpigmentation as common side effects.
    1 context sentence
    Summary · context
    This report presents real-life experiences on the efficacy and safety of setmelanotide, an MC4R agonist, in 4 prepubertal children (ages 3-9) with LEPR and POMC deficiencies.
  3. 03HumanCase report or seriesSafety outcome

    In prepubertal children with LEPR or POMC deficiency, injection site reactions were common side effects of setmelanotide.

    In plain wordsIn prepubertal children with LEPR or POMC deficiency, injection site reactions were common side effects of setmelanotide.

    Peptide
    Setmelanotide
    Outcome
    Not applicableInjection site reactions
    Study

    n = 4

    Outcome group
    Adverse events · Skin and wound
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Treatment was generally well-tolerated, with injection site reactions and hyperpigmentation as common side effects.
    1 context sentence
    Summary · context
    This report presents real-life experiences on the efficacy and safety of setmelanotide, an MC4R agonist, in 4 prepubertal children (ages 3-9) with LEPR and POMC deficiencies.
  4. 04HumanCase report or seriesSafety outcome

    In prepubertal children with LEPR or POMC deficiency, hyperpigmentation was a common side effect of setmelanotide.

    In plain wordsIn prepubertal children with LEPR or POMC deficiency, skin darkening was a common side effect of setmelanotide.

    Peptide
    Setmelanotide
    Outcome
    Not applicableHyperpigmentation
    Study

    n = 4

    Outcome group
    Pigmentation · Skin and wound
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Treatment was generally well-tolerated, with injection site reactions and hyperpigmentation as common side effects.
    1 context sentence
    Summary · context
    This report presents real-life experiences on the efficacy and safety of setmelanotide, an MC4R agonist, in 4 prepubertal children (ages 3-9) with LEPR and POMC deficiencies.
  5. 05HumanCase report or seriesEfficacy outcome

    In a prepubertal child with POMC deficiency receiving setmelanotide, central hypothyroidism resolved.

    In plain wordsIn a prepubertal child with POMC deficiency receiving setmelanotide, central hypothyroidism resolved.

    Peptide
    Setmelanotide
    Outcome
    Not applicableRecovery from central hypothyroidism
    Study

    n = 1

    Outcome group
    Thyroid function · Endocrine and hormones
    Studied dose
    Dose not stated

    Measurements

    Patients with recovery from central hypothyroidism
    1 patient
    Patient with POMC deficiency

    Source sentences

    Summary
    Growth deceleration was noted in 2 children, and recovery from central hypothyroidism in 1 patient with POMC deficiency.
    1 context sentence
    Summary · context
    This report presents real-life experiences on the efficacy and safety of setmelanotide, an MC4R agonist, in 4 prepubertal children (ages 3-9) with LEPR and POMC deficiencies.
  6. 06HumanCase report or seriesOther

    In prepubertal children receiving setmelanotide, gonadotropin-related effects included hypothalamo-pituitary-gonadal axis activation and occurred in 2 patients overall.

    In plain wordsIn prepubertal children receiving setmelanotide, researchers observed activation of the hormone system that controls reproductive development.

    Peptide
    Setmelanotide
    Outcome
    IncreaseHypothalamo-pituitary-gonadal axis activation
    Study

    n: not reported in source

    Outcome group
    Sex hormones and fertility · Endocrine and hormones
    Studied dose
    Dose not stated

    Measurements

    Patients with gonadotropin-related effects
    2 patients
    Effects included HPG axis activation and testicular descent

    Source sentences

    Summary
    As novel findings, gonadotropin-related effects such as hypothalamo-pituitary-gonadal axis activation and testicular descent were observed in 2 patients.
    1 context sentence
    Summary · context
    This report presents real-life experiences on the efficacy and safety of setmelanotide, an MC4R agonist, in 4 prepubertal children (ages 3-9) with LEPR and POMC deficiencies.
  7. 07HumanCase report or seriesOther

    In prepubertal children receiving setmelanotide, reported gonadotropin-related effects included testicular descent; gonadotropin-related effects occurred in 2 patients.

    In plain wordsIn prepubertal children receiving setmelanotide, researchers observed testicular descent among the reported hormone-related effects.

    Peptide
    Setmelanotide
    Outcome
    Not applicableTesticular descent
    Study

    n: not reported in source

    Outcome group
    Sex hormones and fertility · Reproductive and sexual health
    Studied dose
    Dose not stated

    Measurements

    Patients with gonadotropin-related effects
    2 patients
    Effects included HPG axis activation and testicular descent

    Source sentences

    Summary
    As novel findings, gonadotropin-related effects such as hypothalamo-pituitary-gonadal axis activation and testicular descent were observed in 2 patients.
    1 context sentence
    Summary · context
    This report presents real-life experiences on the efficacy and safety of setmelanotide, an MC4R agonist, in 4 prepubertal children (ages 3-9) with LEPR and POMC deficiencies.
  8. 08HumanCase report or seriesSafety outcome

    In prepubertal children receiving setmelanotide, growth deceleration occurred in 2 children.

    In plain wordsIn prepubertal children receiving setmelanotide, growth slowed in 2 children.

    Peptide
    Setmelanotide
    Outcome
    DecreaseGrowth deceleration
    Study

    n: not reported in source

    Outcome group
    Linear growth · Whole body
    Studied dose
    Dose not stated

    Measurements

    Children with growth deceleration
    2 children
    Prepubertal children on setmelanotide

    Source sentences

    Summary
    Growth deceleration was noted in 2 children, and recovery from central hypothyroidism in 1 patient with POMC deficiency.
    1 context sentence
    Summary · context
    This report presents real-life experiences on the efficacy and safety of setmelanotide, an MC4R agonist, in 4 prepubertal children (ages 3-9) with LEPR and POMC deficiencies.

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

  1. Version 2Current

    28 Oct 2026, 02:03 UTC

    Study results updated

  2. Version 1

    27 Oct 2026, 16:36 UTC

    First added · source update from Europe PMC

Subjects

Terms as the source lists them.

Publication types

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