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Study · Case report or series · n = 1 · 2025

6 results

Effectiveness of the Dual GIP/GLP1-Agonist Tirzepatide in 2 Cases of Alström Syndrome, a Rare Obesity Syndrome.

Ferch M, Peitsch I, Kautzky-Willer A, Greber-Platzer S, Stättermayer AF, Krebs M, Scherer T

The Journal of clinical endocrinology and metabolism1 Nov 2025Cited by 7 (Europe PMC)

PMID40302276 (opens in a new tab)PMCIDPMC12623033 (opens in a new tab)DOI10.1210/clinem/dgaf258 (opens in a new tab)PepBankPUB-40302276
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Summary

ContextTirzepatide, a dual glucose-dependent insulinotropic peptide/glucagon-like peptide 1 (GIP/GLP1) receptor agonist, was recently approved for type 2 diabetes and weight management. Alström syndrome (AS) is a rare, genetic, multisystemic disorder, characterized by cone-rod dystrophy, progressive hearing loss, obesity, and diabetes with profound insulin resistance due to marked hyperphagia.ObjectiveHere we highlight the potential of tirzepatide as a novel therapeutic option for improving glycemic outcomes, metabolic dysfunction-associated steatotic liver disease (MASLD), and effectively reducing body weight in individuals with AS.MethodsWe present the first 2 reported cases of people living with AS treated with tirzepatide.ResultsTwo individuals with AS, previously treated with semaglutide, received tirzepatide at our clinic. The first, a 23-year-old man with 18 months of treatment, experienced a weight loss of -28 kg (113.6 to 83 kg, -26.9%); glycated hemoglobin A1c decreased by -0.4% (6.7% to 6.3%), with considerable reductions in daily insulin doses of -96 IU/day (-83%; 58 to 20 IU insulin glargine and 58 to 0 IU prandial insulin), while maintaining oral antidiabetics. Hepatic steatosis, with a previous fat fraction of 20%, resolved as confirmed by magnetic resonance imaging (MRI). The second, a 20-year-old man with previously well-controlled diabetes, was followed up for 9 months and showed a weight reduction of -9.5 kg (132 kg to 122.5 kg; -7.2%) with a reduction of hepatic lipid content from 21% at the latest MRI to 11% after approximately 3 months of therapy.ConclusionTirzepatide shows great effectiveness with regard to body weight, MASLD, and insulin resistance in AS. Follow-up studies with larger cohorts have to be performed to confirm these findings.
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. 01HumanCase report or seriesEfficacy outcome

    In a 23-year-old man with Alström syndrome, tirzepatide for 18 months reduced body weight by 28 kg (-26.9%).

    In plain wordsIn one young man with Alström syndrome, tirzepatide over 18 months was followed by a 28 kg weight loss.

    Peptide
    Tirzepatide
    Outcome
    DecreaseBody weight change, case 1
    Study

    n = 1

    Outcome group
    Body weight · Metabolic and weight
    Studied dose
    Dose not stated · 18 monthsAs given in this study (human).

    Measurements

    Body weight change
    −28 kg
    case 1
    Body weight change
    −26.9%
    case 1

    Source sentences

    Summary
    experienced a weight loss of -28 kg (113.6 to 83 kg, -26.9%)
    1 context sentence
    Summary · context
    Two individuals with AS, previously treated with semaglutide, received tirzepatide at our clinic.
  2. 02HumanCase report or seriesEfficacy outcome

    In a 23-year-old man with Alström syndrome, tirzepatide for 18 months lowered HbA1c by 0.4 percentage points (6.7% to 6.3%).

    In plain wordsIn one young man with Alström syndrome, HbA1c fell from 6.7% to 6.3% after 18 months of tirzepatide.

    Peptide
    Tirzepatide
    Outcome
    DecreaseHbA1c change, case 1
    Study

    n = 1

    Outcome group
    Glycemic control · Metabolic and weight
    Studied dose
    Dose not stated · 18 monthsAs given in this study (human).

    Measurements

    HbA1c change
    −0.4%
    case 1

    Source sentences

    Summary
    glycated hemoglobin A1c decreased by -0.4% (6.7% to 6.3%)
    1 context sentence
    Summary · context
    Two individuals with AS, previously treated with semaglutide, received tirzepatide at our clinic.
  3. 03HumanCase report or seriesEfficacy outcome

    In a 23-year-old man with Alström syndrome, tirzepatide for 18 months allowed a reduction in daily insulin dose of 96 IU (83%), while he stayed on oral antidiabetics.

    In plain wordsIn one young man with Alström syndrome, daily insulin needs fell by 83% after 18 months of tirzepatide.

    Peptide
    Tirzepatide
    Outcome
    DecreaseDaily insulin dose, case 1
    Study

    n = 1

    Outcome group
    Glycemic control · Metabolic and weight
    Studied dose
    Dose not stated · 18 monthsAs given in this study (human).

    Measurements

    Daily insulin dose change
    −96 IU/day
    case 1
    Daily insulin dose change
    −83%
    case 1

    Source sentences

    Summary
    with considerable reductions in daily insulin doses of -96 IU/day (-83%; 58 to 20 IU insulin glargine and 58 to 0 IU prandial insulin)
    1 context sentence
    Summary · context
    Two individuals with AS, previously treated with semaglutide, received tirzepatide at our clinic.
  4. 04HumanCase report or seriesEfficacy outcome

    In the same 23-year-old man, hepatic steatosis with a previous fat fraction of 20% resolved on MRI during tirzepatide treatment.

    In plain wordsIn this young man with Alström syndrome, fatty liver seen before treatment was gone on MRI after tirzepatide.

    Peptide
    Tirzepatide
    Outcome
    DecreaseHepatic steatosis (MRI fat fraction), case 1
    Study

    n = 1

    Outcome group
    Liver fat and liver function · Liver
    Studied dose
    Dose not stated · 18 monthsAs given in this study (human).

    Measurements

    Hepatic fat fraction (previous)
    20%
    case 1

    Source sentences

    Summary
    Hepatic steatosis, with a previous fat fraction of 20%, resolved as confirmed by magnetic resonance imaging (MRI).
    1 context sentence
    Summary · context
    Two individuals with AS, previously treated with semaglutide, received tirzepatide at our clinic.
  5. 05HumanCase report or seriesEfficacy outcome

    In a 20-year-old man with Alström syndrome and previously well-controlled diabetes, tirzepatide for 9 months reduced body weight by 9.5 kg (-7.2%).

    In plain wordsIn a second young man with Alström syndrome, tirzepatide over 9 months was followed by a 9.5 kg weight loss.

    Peptide
    Tirzepatide
    Outcome
    DecreaseBody weight change, case 2
    Study

    n = 1

    Outcome group
    Body weight · Metabolic and weight
    Studied dose
    Dose not stated · 9 monthsAs given in this study (human).

    Measurements

    Body weight change
    −9.5 kg
    case 2
    Body weight change
    −7.2%
    case 2

    Source sentences

    Summary
    showed a weight reduction of -9.5 kg (132 kg to 122.5 kg; -7.2%)
    1 context sentence
    Summary · context
    Two individuals with AS, previously treated with semaglutide, received tirzepatide at our clinic.
  6. 06HumanCase report or seriesEfficacy outcome

    In the same 20-year-old man, hepatic lipid content fell from 21% to 11% after approximately 3 months of tirzepatide.

    In plain wordsIn this second young man, liver fat fell from 21% to 11% after about 3 months of tirzepatide.

    Peptide
    Tirzepatide
    Outcome
    DecreaseHepatic lipid content, case 2
    Study

    n = 1

    Outcome group
    Liver fat and liver function · Liver
    Studied dose
    Dose not stated · approximately 3 monthsAs given in this study (human).

    Measurements

    Hepatic lipid content
    21%
    latest MRI before
    Hepatic lipid content
    11%
    after ~3 months

    Source sentences

    Summary
    a reduction of hepatic lipid content from 21% at the latest MRI to 11% after approximately 3 months of therapy.
    1 context sentence
    Summary · context
    Two individuals with AS, previously treated with semaglutide, received tirzepatide at our clinic.

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 3Current

    11 Oct 2026, 00:04 UTC

    Study results updated

  2. Version 2

    9 Oct 2026, 03:07 UTC

    Study data updated · source update from Europe PMC

  3. Version 1

    9 Oct 2026, 03:06 UTC

    First added · source update from Europe PMC

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Terms as the source lists them.

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