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Study · Observational study · 2025

6 results

Patient and caregiver experiences with a patient-support program for setmelanotide treatment of patients with Bardet-Biedl syndrome.

Finkelberg I, Polichronidou IM, Hühne T, Brensing P, Karaterzi S, Jaegers J, Gäckler A, Pape L, Cetiner M

Orphanet journal of rare diseases8 Jun 2025Cited by 9 (Europe PMC)

PMID40484968 (opens in a new tab)PMCIDPMC12147271 (opens in a new tab)DOI10.1186/s13023-025-03835-9 (opens in a new tab)PepBankPUB-40484968
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Summary

BackgroundBardet-Biedl syndrome (BBS) is a rare genetic disease caused by impaired cilium function and characterized by a plethora of symptoms, including hyperphagia and early-onset obesity, that negatively affect patient and caregiver quality of life. Here, we assessed real-world patient expectations and experiences before and during treatment with setmelanotide, a melanocortin-4 receptor agonist shown to reduce hunger and body weight in patients with BBS.MethodsAn online survey was conducted to capture the real-world experience of patients with BBS and their caregivers regarding setmelanotide treatment and the use of a specialist nurse support service aimed at educating patients and their caregivers, and enabling them to administer injections independently. The survey was administered between January 2024 and May 2024 to participants who began treatment between June 2023 and December 2023 at a single center in Germany.ResultsOf the 35 respondents, 10 were pediatric patients, 13 were adult patients, and 12 were caregivers. Prior to treatment, the most commonly reported symptoms by pediatric patients and caregivers were insatiable hunger (80% and 83%, respectively) and obesity (50% and 92%, respectively); for adult patients, key symptoms were vision loss (92%) and obesity (69%). Setmelanotide reduced feelings of insatiable hunger and had a positive effect on body weight: ≥ 92% of respondents across survey groups reported feeling less hunger, feeling satiated after meals, and a stable body weight or weight loss (mean BMI z-score ± SD at start: 3.12 ± 0.89, change after 6 months: -0.47 ± 0.37). Improvements in mobility, mood, and behavior were also reported. The specialist nurse support service was rated excellent by all respondents. The personalized approach contributed to high patient and caregiver satisfaction, enabled most of them to administer the drug independently, and ensured high treatment adherence, without any patients discontinuing setmelanotide treatment.ConclusionBased on this real-world survey of patients with BBS and their caregivers, setmelanotide improved key symptoms related to insatiable hunger and obesity. Personalized multidimensional nursing support at the start of treatment can help address unmet support needs in BBS and may contribute to high rates of treatment satisfaction and adherence.
CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study6

6 results from studies that gave the peptide. 6 results from people.

  1. 01HumanObservational studyEfficacy outcome

    In a BBS survey, at least 92% across respondent groups reported less hunger during setmelanotide treatment.

    In plain wordsIn a BBS survey, setmelanotide treatment was linked to less hunger in at least 92% across respondent groups.

    Peptide
    Setmelanotide
    Outcome
    DecreaseInsatiable hunger
    Study

    n: not reported in source

    Outcome group
    Appetite and food intake · Metabolic and weight
    Studied dose
    Dose not stated

    Measurements

    Respondents reporting less hunger
    ≥ 92%
    Across survey groups

    Source sentences

    Summary
    Setmelanotide reduced feelings of insatiable hunger and had a positive effect on body weight: ≥ 92% of respondents across survey groups reported feeling less hunger, feeling satiated after meals, and a stable body weight or weight loss (mean BMI z-score ± SD at start: 3.12 ± 0.89, change after 6 months: -0.47 ± 0.37).
    1 context sentence
    Summary · context
    An online survey was conducted to capture the real-world experience of patients with BBS and their caregivers regarding setmelanotide treatment and the use of a specialist nurse support service aimed at educating patients and their caregivers, and enabling them to administer injections independently.
  2. 02HumanObservational studyEfficacy outcome

    In a BBS survey, at least 92% across respondent groups reported satiety after meals during setmelanotide treatment.

    In plain wordsIn a BBS survey, setmelanotide treatment was linked to feeling full after meals in at least 92% across respondent groups.

    Peptide
    Setmelanotide
    Outcome
    IncreaseSatiety after meals
    Study

    n: not reported in source

    Outcome group
    Appetite and food intake · Metabolic and weight
    Studied dose
    Dose not stated

    Measurements

    Respondents reporting satiety after meals
    ≥ 92%
    Across survey groups

    Source sentences

    Summary
    Setmelanotide reduced feelings of insatiable hunger and had a positive effect on body weight: ≥ 92% of respondents across survey groups reported feeling less hunger, feeling satiated after meals, and a stable body weight or weight loss (mean BMI z-score ± SD at start: 3.12 ± 0.89, change after 6 months: -0.47 ± 0.37).
    1 context sentence
    Summary · context
    An online survey was conducted to capture the real-world experience of patients with BBS and their caregivers regarding setmelanotide treatment and the use of a specialist nurse support service aimed at educating patients and their caregivers, and enabling them to administer injections independently.
  3. 03HumanObservational studyEfficacy outcome

    In a BBS survey, at least 92% across respondent groups reported stable weight or weight loss during setmelanotide treatment.

    In plain wordsIn a BBS survey, setmelanotide treatment was linked to stable weight or weight loss in at least 92% across respondent groups.

    Peptide
    Setmelanotide
    Outcome
    MixedBody weight stability or loss
    Study

    n: not reported in source

    Outcome group
    Body weight · Metabolic and weight
    Studied dose
    Dose not stated

    Measurements

    Respondents reporting stable weight or weight loss
    ≥ 92%
    Across survey groups

    Source sentences

    Summary
    Setmelanotide reduced feelings of insatiable hunger and had a positive effect on body weight: ≥ 92% of respondents across survey groups reported feeling less hunger, feeling satiated after meals, and a stable body weight or weight loss (mean BMI z-score ± SD at start: 3.12 ± 0.89, change after 6 months: -0.47 ± 0.37).
    1 context sentence
    Summary · context
    An online survey was conducted to capture the real-world experience of patients with BBS and their caregivers regarding setmelanotide treatment and the use of a specialist nurse support service aimed at educating patients and their caregivers, and enabling them to administer injections independently.
  4. 04HumanObservational studyEfficacy outcome

    In patients with BBS receiving setmelanotide, mean BMI z-score changed by -0.47 ± 0.37 after 6 months from 3.12 ± 0.89.

    In plain wordsIn patients with BBS, setmelanotide treatment was linked to a lower BMI z-score after 6 months.

    Peptide
    Setmelanotide
    Outcome
    DecreaseBMI z-score change
    Study

    n: not reported in source

    Outcome group
    Body weight · Metabolic and weight
    Studied dose
    Dose not stated

    Measurements

    Baseline mean BMI z-score
    3.12 z-score
    At treatment start
    Baseline BMI z-score SD
    0.89 z-score
    At treatment start
    Mean BMI z-score change
    −0.47 z-score
    After 6 months
    BMI z-score change SD
    0.37 z-score
    After 6 months

    Source sentences

    Summary
    Setmelanotide reduced feelings of insatiable hunger and had a positive effect on body weight: ≥ 92% of respondents across survey groups reported feeling less hunger, feeling satiated after meals, and a stable body weight or weight loss (mean BMI z-score ± SD at start: 3.12 ± 0.89, change after 6 months: -0.47 ± 0.37).
    1 context sentence
    Summary · context
    An online survey was conducted to capture the real-world experience of patients with BBS and their caregivers regarding setmelanotide treatment and the use of a specialist nurse support service aimed at educating patients and their caregivers, and enabling them to administer injections independently.
  5. 05HumanObservational studyOther

    In the BBS survey, no patients discontinued setmelanotide during the nurse-supported treatment program.

    In plain wordsIn the BBS survey, no patients stopped setmelanotide during the nurse-supported treatment program.

    Peptide
    Setmelanotide
    Outcome
    Not applicableTreatment discontinuation
    Study

    n: not reported in source

    Outcome group
    Treatment adherence and retention · Whole body
    Studied dose
    Dose not stated

    Source sentences

    Summary
    The personalized approach contributed to high patient and caregiver satisfaction, enabled most of them to administer the drug independently, and ensured high treatment adherence, without any patients discontinuing setmelanotide treatment.
    1 context sentence
    Summary · context
    An online survey was conducted to capture the real-world experience of patients with BBS and their caregivers regarding setmelanotide treatment and the use of a specialist nurse support service aimed at educating patients and their caregivers, and enabling them to administer injections independently.
  6. 06HumanObservational studyEfficacy outcome

    In the BBS survey during setmelanotide treatment, respondents reported improvements in mobility, mood, and behavior.

    In plain wordsIn the BBS survey, setmelanotide treatment was linked to reported improvements in mobility, mood, and behavior.

    Peptide
    Setmelanotide
    Outcome
    Not applicableReported mobility, mood, and behavior
    Study

    n: not reported in source

    Outcome group
    Quality of life · Whole body
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Improvements in mobility, mood, and behavior were also reported.
    1 context sentence
    Summary · context
    An online survey was conducted to capture the real-world experience of patients with BBS and their caregivers regarding setmelanotide treatment and the use of a specialist nurse support service aimed at educating patients and their caregivers, and enabling them to administer injections independently.

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.

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