Alina Z Mehdi, Lily Deng, Colby L Chase, Maria Cristina Foss-Freitas, Brigid Gregg, Rochelle N Naylor, Elif A Oral, William H Herman, Mansa Krishnamurthy, David T Broome
{"title":"MODY的GLP-1 RA和双重GIP/GLP-1 RA治疗：描述性病例系列。","authors":"Alina Z Mehdi, Lily Deng, Colby L Chase, Maria Cristina Foss-Freitas, Brigid Gregg, Rochelle N Naylor, Elif A Oral, William H Herman, Mansa Krishnamurthy, David T Broome","doi":"10.1136/bmjdrc-2024-004885","DOIUrl":null,"url":null,"abstract":"<p><strong>Introduction: </strong>Glucagon-like peptide-1 receptor agonists (GLP-1 RA) and dual glucose insulinotropic polypeptide (GIP)/GLP-1 RA are widely prescribed, but their effectiveness in different subtypes of maturity-onset diabetes of the young (MODY) is unknown.</p><p><strong>Research design and methods: </strong>We present a descriptive case series of individuals from two MODY cohorts who used GLP-1 RA or dual GIP/GLP-1 RA. Paired <i>t</i> tests were used to compare HbA1c, body mass index (BMI), and sulfonylurea (SU) dose before and after GLP-1 RA or dual GIP/GLP-1 RA therapy.</p><p><strong>Results: </strong>10 individuals (4 hepatocyte nuclear factor-1α (HNF1A)-MODY, 4 hepatocyte nuclear factor-4α (HNF4A)-MODY, 1 ATP-binding cassette transporter subfamily C member 8 (ABCC8)-MODY, 1 hepatocyte nuclear factor-1β (HNF1B)-MODY) were identified who used GLP-1 RA or dual GIP/GLP-1 RA. In patients with HNF1A-MODY and HNF4A-MODY, GLP-1 RA reduced HbA1c by 1.3% (p=0.010), BMI by 2.90 kg/m<sup>2</sup> (p=0.008), and total daily dose of SU by 66.6% (p=0.005). Dual GIP/GLP-1 RA treatment led to a non-statistically significant decrease in HbA1c of 1.8% (p=0.104), a statistically significant reduction in BMI of 8.73 kg/m<sup>2</sup> (p=0.010), and all patients discontinued SU (n=2) and one discontinued insulin. In patients with ABCC8-MODY and HNF1B-MODY, GLP-1 RA reduced HbA1c by 1.2% and 1.8%, BMI by 1.1 kg/m<sup>2</sup> and 1.2 kg/m<sup>2</sup>, and the patients no longer required treatment with SU or insulin, respectively.</p><p><strong>Conclusions: </strong>GLP-1 RA and dual GIP/GLP-1 RA lowered HbA1c, BMI, and SU dose in patients with MODY.</p>","PeriodicalId":9151,"journal":{"name":"BMJ Open Diabetes Research & Care","volume":"13 2","pages":""},"PeriodicalIF":3.7000,"publicationDate":"2025-04-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12020758/pdf/","citationCount":"0","resultStr":"{\"title\":\"GLP-1 RA and dual GIP/GLP-1 RA treatment in MODY: a descriptive case series.\",\"authors\":\"Alina Z Mehdi, Lily Deng, Colby L Chase, Maria Cristina Foss-Freitas, Brigid Gregg, Rochelle N Naylor, Elif A Oral, William H Herman, Mansa Krishnamurthy, David T Broome\",\"doi\":\"10.1136/bmjdrc-2024-004885\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Introduction: </strong>Glucagon-like peptide-1 receptor agonists (GLP-1 RA) and dual glucose insulinotropic polypeptide (GIP)/GLP-1 RA are widely prescribed, but their effectiveness in different subtypes of maturity-onset diabetes of the young (MODY) is unknown.</p><p><strong>Research design and methods: </strong>We present a descriptive case series of individuals from two MODY cohorts who used GLP-1 RA or dual GIP/GLP-1 RA. Paired <i>t</i> tests were used to compare HbA1c, body mass index (BMI), and sulfonylurea (SU) dose before and after GLP-1 RA or dual GIP/GLP-1 RA therapy.</p><p><strong>Results: </strong>10 individuals (4 hepatocyte nuclear factor-1α (HNF1A)-MODY, 4 hepatocyte nuclear factor-4α (HNF4A)-MODY, 1 ATP-binding cassette transporter subfamily C member 8 (ABCC8)-MODY, 1 hepatocyte nuclear factor-1β (HNF1B)-MODY) were identified who used GLP-1 RA or dual GIP/GLP-1 RA. In patients with HNF1A-MODY and HNF4A-MODY, GLP-1 RA reduced HbA1c by 1.3% (p=0.010), BMI by 2.90 kg/m<sup>2</sup> (p=0.008), and total daily dose of SU by 66.6% (p=0.005). Dual GIP/GLP-1 RA treatment led to a non-statistically significant decrease in HbA1c of 1.8% (p=0.104), a statistically significant reduction in BMI of 8.73 kg/m<sup>2</sup> (p=0.010), and all patients discontinued SU (n=2) and one discontinued insulin. In patients with ABCC8-MODY and HNF1B-MODY, GLP-1 RA reduced HbA1c by 1.2% and 1.8%, BMI by 1.1 kg/m<sup>2</sup> and 1.2 kg/m<sup>2</sup>, and the patients no longer required treatment with SU or insulin, respectively.</p><p><strong>Conclusions: </strong>GLP-1 RA and dual GIP/GLP-1 RA lowered HbA1c, BMI, and SU dose in patients with MODY.</p>\",\"PeriodicalId\":9151,\"journal\":{\"name\":\"BMJ Open Diabetes Research & Care\",\"volume\":\"13 2\",\"pages\":\"\"},\"PeriodicalIF\":3.7000,\"publicationDate\":\"2025-04-23\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12020758/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"BMJ Open Diabetes Research & Care\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1136/bmjdrc-2024-004885\",\"RegionNum\":2,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"ENDOCRINOLOGY & METABOLISM\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"BMJ Open Diabetes Research & Care","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1136/bmjdrc-2024-004885","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"ENDOCRINOLOGY & METABOLISM","Score":null,"Total":0}
GLP-1 RA and dual GIP/GLP-1 RA treatment in MODY: a descriptive case series.
Introduction: Glucagon-like peptide-1 receptor agonists (GLP-1 RA) and dual glucose insulinotropic polypeptide (GIP)/GLP-1 RA are widely prescribed, but their effectiveness in different subtypes of maturity-onset diabetes of the young (MODY) is unknown.
Research design and methods: We present a descriptive case series of individuals from two MODY cohorts who used GLP-1 RA or dual GIP/GLP-1 RA. Paired t tests were used to compare HbA1c, body mass index (BMI), and sulfonylurea (SU) dose before and after GLP-1 RA or dual GIP/GLP-1 RA therapy.
Results: 10 individuals (4 hepatocyte nuclear factor-1α (HNF1A)-MODY, 4 hepatocyte nuclear factor-4α (HNF4A)-MODY, 1 ATP-binding cassette transporter subfamily C member 8 (ABCC8)-MODY, 1 hepatocyte nuclear factor-1β (HNF1B)-MODY) were identified who used GLP-1 RA or dual GIP/GLP-1 RA. In patients with HNF1A-MODY and HNF4A-MODY, GLP-1 RA reduced HbA1c by 1.3% (p=0.010), BMI by 2.90 kg/m2 (p=0.008), and total daily dose of SU by 66.6% (p=0.005). Dual GIP/GLP-1 RA treatment led to a non-statistically significant decrease in HbA1c of 1.8% (p=0.104), a statistically significant reduction in BMI of 8.73 kg/m2 (p=0.010), and all patients discontinued SU (n=2) and one discontinued insulin. In patients with ABCC8-MODY and HNF1B-MODY, GLP-1 RA reduced HbA1c by 1.2% and 1.8%, BMI by 1.1 kg/m2 and 1.2 kg/m2, and the patients no longer required treatment with SU or insulin, respectively.
Conclusions: GLP-1 RA and dual GIP/GLP-1 RA lowered HbA1c, BMI, and SU dose in patients with MODY.
期刊介绍：
BMJ Open Diabetes Research & Care is an open access journal committed to publishing high-quality, basic and clinical research articles regarding type 1 and type 2 diabetes, and associated complications. Only original content will be accepted, and submissions are subject to rigorous peer review to ensure the publication of
high-quality — and evidence-based — original research articles.
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