Longitudinal progression of diabetes mellitus in Wolfram syndrome: The Washington University Wolfram Research Clinic experience
Objective (1) Describe the progression of diabetes mellitus over time in an observational study of Wolfram syndrome, a rare, genetic, neurodegenerative disorder, which often includes diabetes mellitus and is typically diagnosed during childhood or adolescence. (2) Determine whether C‐peptide could b...
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| Veröffentlicht in: | Pediatric diabetes 2022-03, Vol.23 (2), p.212-218 |
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| creator | Ray, Mary Katherine Chen, Ling White, Neil H. Ni, Richard Hershey, Tamara Marshall, Bess A. |
| description | Objective
(1) Describe the progression of diabetes mellitus over time in an observational study of Wolfram syndrome, a rare, genetic, neurodegenerative disorder, which often includes diabetes mellitus and is typically diagnosed during childhood or adolescence. (2) Determine whether C‐peptide could be used as a marker of diabetes progression in interventional trials for Wolfram syndrome.
Methods
N = 44 (25F/19M) participants with genetically confirmed Wolfram syndrome attended the Washington University Wolfram Research Clinic annually from 2010 to 2019. Medical history, physical examinations, blood sampling, and questionnaires were used to collect data about diabetes mellitus and other components of Wolfram syndrome. Beta‐cell function was assessed by determination of C‐peptide during a mixed meal tolerance test. Random coefficients models evaluated the rate of progression of C‐peptide over time, and power analyses were used to estimate the number of subjects needed to detect a change in C‐peptide decline during an intervention trial.
Results
93.2% of patients had diabetes mellitus. Mean HbA1c across all study visits was 7.9%. C‐peptide significantly decreased with increasing duration of diabetes mellitus (p |
| doi_str_mv | 10.1111/pedi.13291 |
| format | Article |
| fullrecord | <record><control><sourceid>proquest_pubme</sourceid><recordid>TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_8844189</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><sourcerecordid>2628239239</sourcerecordid><originalsourceid>FETCH-LOGICAL-c5141-1c1920f830d968a641ff0bb70db9baee083935332f4763c70e026d33c9b22fa83</originalsourceid><addsrcrecordid>eNp9kV1rFDEUhoMotlZv_AES8EYKW_M1M4kXgmyrFhYUaal3IZM52U2ZSabJTHWv_Oumbl3UC0MgB86Th8N5EXpOyQkt5_UInT-hnCn6AB1SrtSiEkI-3Nf86wF6kvM1IbRRXDxGB1w0irG6OUQ_VjGs_TR3PpgejymuE-TsY8DR4c6bFibIeIC-L1DGPuCr2LtkBpy3oUtxgDf4YgP4yuSND-upfLwM_hZS9tN2z36BDCbZDV72PniL4fsIyUOw8BQ9cqbP8Oz-PUKX788ulh8Xq08fzpfvVgtbUUEX1FLFiJOcdKqWphbUOdK2Dela1RoAIrniFefMiabmtiFAWN1xblXLmDOSH6G3O-84twN0FsKUTK_H5AeTtjoar__uBL_R63irpRSCSlUEr-4FKd7MkCc9-GzLXkyAOGfNKqVIIyoiCvryH_Q6zqnst1A1k4yrcgt1vKNsijkncPthKNF3ueq7XPWvXAv84s_x9-jvIAtAd8A338P2Pyr9-ez0fCf9CTYRsKo</addsrcrecordid><sourcetype>Open Access Repository</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype><pqid>2628239239</pqid></control><display><type>article</type><title>Longitudinal progression of diabetes mellitus in Wolfram syndrome: The Washington University Wolfram Research Clinic experience</title><source>MEDLINE</source><source>Wiley Online Library All Journals</source><creator>Ray, Mary Katherine ; Chen, Ling ; White, Neil H. ; Ni, Richard ; Hershey, Tamara ; Marshall, Bess A.</creator><creatorcontrib>Ray, Mary Katherine ; Chen, Ling ; White, Neil H. ; Ni, Richard ; Hershey, Tamara ; Marshall, Bess A.</creatorcontrib><description>Objective
(1) Describe the progression of diabetes mellitus over time in an observational study of Wolfram syndrome, a rare, genetic, neurodegenerative disorder, which often includes diabetes mellitus and is typically diagnosed during childhood or adolescence. (2) Determine whether C‐peptide could be used as a marker of diabetes progression in interventional trials for Wolfram syndrome.
Methods
N = 44 (25F/19M) participants with genetically confirmed Wolfram syndrome attended the Washington University Wolfram Research Clinic annually from 2010 to 2019. Medical history, physical examinations, blood sampling, and questionnaires were used to collect data about diabetes mellitus and other components of Wolfram syndrome. Beta‐cell function was assessed by determination of C‐peptide during a mixed meal tolerance test. Random coefficients models evaluated the rate of progression of C‐peptide over time, and power analyses were used to estimate the number of subjects needed to detect a change in C‐peptide decline during an intervention trial.
Results
93.2% of patients had diabetes mellitus. Mean HbA1c across all study visits was 7.9%. C‐peptide significantly decreased with increasing duration of diabetes mellitus (p < 0.0001); an optimal break point in C‐peptide decline was identified to occur between 0.1 and 2.3 years after diabetes mellitus diagnosis. Twenty patients per group (active vs. control) were estimated to be needed to detect a 60% slowing of C‐peptide decline during the first 2.3 years following diabetes diagnosis.
Conclusion
C‐peptide declines over time in Wolfram syndrome and could potentially be used as a marker of diabetes progression in interventional studies for Wolfram syndrome, especially within the first 2 years after diabetes diagnosis.</description><identifier>ISSN: 1399-543X</identifier><identifier>EISSN: 1399-5448</identifier><identifier>DOI: 10.1111/pedi.13291</identifier><identifier>PMID: 34792267</identifier><language>eng</language><publisher>Former Munksgaard: John Wiley & Sons A/S</publisher><subject>Adolescent ; Ambulatory Care Facilities - organization & administration ; Ambulatory Care Facilities - statistics & numerical data ; Chi-Square Distribution ; Child ; Children ; Clinical trials ; C‐peptide ; Diabetes ; Diabetes mellitus ; Diabetes Mellitus - epidemiology ; Diabetes Mellitus - etiology ; Diagnosis ; DIDMOAD ; Disease Progression ; Female ; Hb A1c ; Humans ; Longitudinal Studies ; Male ; neurodegenerative disease ; Neurodegenerative diseases ; Patients ; Peptides ; Washington - epidemiology ; Wolfram Syndrome - complications ; Wolfram Syndrome - epidemiology</subject><ispartof>Pediatric diabetes, 2022-03, Vol.23 (2), p.212-218</ispartof><rights>2021 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd</rights><rights>2021 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.</rights><rights>2022 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c5141-1c1920f830d968a641ff0bb70db9baee083935332f4763c70e026d33c9b22fa83</citedby><cites>FETCH-LOGICAL-c5141-1c1920f830d968a641ff0bb70db9baee083935332f4763c70e026d33c9b22fa83</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://onlinelibrary.wiley.com/doi/pdf/10.1111%2Fpedi.13291$$EPDF$$P50$$Gwiley$$H</linktopdf><linktohtml>$$Uhttps://onlinelibrary.wiley.com/doi/full/10.1111%2Fpedi.13291$$EHTML$$P50$$Gwiley$$H</linktohtml><link.rule.ids>230,314,780,784,885,1417,27924,27925,45574,45575</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/34792267$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Ray, Mary Katherine</creatorcontrib><creatorcontrib>Chen, Ling</creatorcontrib><creatorcontrib>White, Neil H.</creatorcontrib><creatorcontrib>Ni, Richard</creatorcontrib><creatorcontrib>Hershey, Tamara</creatorcontrib><creatorcontrib>Marshall, Bess A.</creatorcontrib><title>Longitudinal progression of diabetes mellitus in Wolfram syndrome: The Washington University Wolfram Research Clinic experience</title><title>Pediatric diabetes</title><addtitle>Pediatr Diabetes</addtitle><description>Objective
(1) Describe the progression of diabetes mellitus over time in an observational study of Wolfram syndrome, a rare, genetic, neurodegenerative disorder, which often includes diabetes mellitus and is typically diagnosed during childhood or adolescence. (2) Determine whether C‐peptide could be used as a marker of diabetes progression in interventional trials for Wolfram syndrome.
Methods
N = 44 (25F/19M) participants with genetically confirmed Wolfram syndrome attended the Washington University Wolfram Research Clinic annually from 2010 to 2019. Medical history, physical examinations, blood sampling, and questionnaires were used to collect data about diabetes mellitus and other components of Wolfram syndrome. Beta‐cell function was assessed by determination of C‐peptide during a mixed meal tolerance test. Random coefficients models evaluated the rate of progression of C‐peptide over time, and power analyses were used to estimate the number of subjects needed to detect a change in C‐peptide decline during an intervention trial.
Results
93.2% of patients had diabetes mellitus. Mean HbA1c across all study visits was 7.9%. C‐peptide significantly decreased with increasing duration of diabetes mellitus (p < 0.0001); an optimal break point in C‐peptide decline was identified to occur between 0.1 and 2.3 years after diabetes mellitus diagnosis. Twenty patients per group (active vs. control) were estimated to be needed to detect a 60% slowing of C‐peptide decline during the first 2.3 years following diabetes diagnosis.
Conclusion
C‐peptide declines over time in Wolfram syndrome and could potentially be used as a marker of diabetes progression in interventional studies for Wolfram syndrome, especially within the first 2 years after diabetes diagnosis.</description><subject>Adolescent</subject><subject>Ambulatory Care Facilities - organization & administration</subject><subject>Ambulatory Care Facilities - statistics & numerical data</subject><subject>Chi-Square Distribution</subject><subject>Child</subject><subject>Children</subject><subject>Clinical trials</subject><subject>C‐peptide</subject><subject>Diabetes</subject><subject>Diabetes mellitus</subject><subject>Diabetes Mellitus - epidemiology</subject><subject>Diabetes Mellitus - etiology</subject><subject>Diagnosis</subject><subject>DIDMOAD</subject><subject>Disease Progression</subject><subject>Female</subject><subject>Hb A1c</subject><subject>Humans</subject><subject>Longitudinal Studies</subject><subject>Male</subject><subject>neurodegenerative disease</subject><subject>Neurodegenerative diseases</subject><subject>Patients</subject><subject>Peptides</subject><subject>Washington - epidemiology</subject><subject>Wolfram Syndrome - complications</subject><subject>Wolfram Syndrome - epidemiology</subject><issn>1399-543X</issn><issn>1399-5448</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2022</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNp9kV1rFDEUhoMotlZv_AES8EYKW_M1M4kXgmyrFhYUaal3IZM52U2ZSabJTHWv_Oumbl3UC0MgB86Th8N5EXpOyQkt5_UInT-hnCn6AB1SrtSiEkI-3Nf86wF6kvM1IbRRXDxGB1w0irG6OUQ_VjGs_TR3PpgejymuE-TsY8DR4c6bFibIeIC-L1DGPuCr2LtkBpy3oUtxgDf4YgP4yuSND-upfLwM_hZS9tN2z36BDCbZDV72PniL4fsIyUOw8BQ9cqbP8Oz-PUKX788ulh8Xq08fzpfvVgtbUUEX1FLFiJOcdKqWphbUOdK2Dela1RoAIrniFefMiabmtiFAWN1xblXLmDOSH6G3O-84twN0FsKUTK_H5AeTtjoar__uBL_R63irpRSCSlUEr-4FKd7MkCc9-GzLXkyAOGfNKqVIIyoiCvryH_Q6zqnst1A1k4yrcgt1vKNsijkncPthKNF3ueq7XPWvXAv84s_x9-jvIAtAd8A338P2Pyr9-ez0fCf9CTYRsKo</recordid><startdate>202203</startdate><enddate>202203</enddate><creator>Ray, Mary Katherine</creator><creator>Chen, Ling</creator><creator>White, Neil H.</creator><creator>Ni, Richard</creator><creator>Hershey, Tamara</creator><creator>Marshall, Bess A.</creator><general>John Wiley & Sons A/S</general><general>Wiley Subscription Services, Inc</general><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7T5</scope><scope>H94</scope><scope>K9.</scope><scope>NAPCQ</scope><scope>7X8</scope><scope>5PM</scope></search><sort><creationdate>202203</creationdate><title>Longitudinal progression of diabetes mellitus in Wolfram syndrome: The Washington University Wolfram Research Clinic experience</title><author>Ray, Mary Katherine ; Chen, Ling ; White, Neil H. ; Ni, Richard ; Hershey, Tamara ; Marshall, Bess A.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c5141-1c1920f830d968a641ff0bb70db9baee083935332f4763c70e026d33c9b22fa83</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2022</creationdate><topic>Adolescent</topic><topic>Ambulatory Care Facilities - organization & administration</topic><topic>Ambulatory Care Facilities - statistics & numerical data</topic><topic>Chi-Square Distribution</topic><topic>Child</topic><topic>Children</topic><topic>Clinical trials</topic><topic>C‐peptide</topic><topic>Diabetes</topic><topic>Diabetes mellitus</topic><topic>Diabetes Mellitus - epidemiology</topic><topic>Diabetes Mellitus - etiology</topic><topic>Diagnosis</topic><topic>DIDMOAD</topic><topic>Disease Progression</topic><topic>Female</topic><topic>Hb A1c</topic><topic>Humans</topic><topic>Longitudinal Studies</topic><topic>Male</topic><topic>neurodegenerative disease</topic><topic>Neurodegenerative diseases</topic><topic>Patients</topic><topic>Peptides</topic><topic>Washington - epidemiology</topic><topic>Wolfram Syndrome - complications</topic><topic>Wolfram Syndrome - epidemiology</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Ray, Mary Katherine</creatorcontrib><creatorcontrib>Chen, Ling</creatorcontrib><creatorcontrib>White, Neil H.</creatorcontrib><creatorcontrib>Ni, Richard</creatorcontrib><creatorcontrib>Hershey, Tamara</creatorcontrib><creatorcontrib>Marshall, Bess A.</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>Immunology Abstracts</collection><collection>AIDS and Cancer Research Abstracts</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>Nursing & Allied Health Premium</collection><collection>MEDLINE - Academic</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>Pediatric diabetes</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Ray, Mary Katherine</au><au>Chen, Ling</au><au>White, Neil H.</au><au>Ni, Richard</au><au>Hershey, Tamara</au><au>Marshall, Bess A.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Longitudinal progression of diabetes mellitus in Wolfram syndrome: The Washington University Wolfram Research Clinic experience</atitle><jtitle>Pediatric diabetes</jtitle><addtitle>Pediatr Diabetes</addtitle><date>2022-03</date><risdate>2022</risdate><volume>23</volume><issue>2</issue><spage>212</spage><epage>218</epage><pages>212-218</pages><issn>1399-543X</issn><eissn>1399-5448</eissn><abstract>Objective
(1) Describe the progression of diabetes mellitus over time in an observational study of Wolfram syndrome, a rare, genetic, neurodegenerative disorder, which often includes diabetes mellitus and is typically diagnosed during childhood or adolescence. (2) Determine whether C‐peptide could be used as a marker of diabetes progression in interventional trials for Wolfram syndrome.
Methods
N = 44 (25F/19M) participants with genetically confirmed Wolfram syndrome attended the Washington University Wolfram Research Clinic annually from 2010 to 2019. Medical history, physical examinations, blood sampling, and questionnaires were used to collect data about diabetes mellitus and other components of Wolfram syndrome. Beta‐cell function was assessed by determination of C‐peptide during a mixed meal tolerance test. Random coefficients models evaluated the rate of progression of C‐peptide over time, and power analyses were used to estimate the number of subjects needed to detect a change in C‐peptide decline during an intervention trial.
Results
93.2% of patients had diabetes mellitus. Mean HbA1c across all study visits was 7.9%. C‐peptide significantly decreased with increasing duration of diabetes mellitus (p < 0.0001); an optimal break point in C‐peptide decline was identified to occur between 0.1 and 2.3 years after diabetes mellitus diagnosis. Twenty patients per group (active vs. control) were estimated to be needed to detect a 60% slowing of C‐peptide decline during the first 2.3 years following diabetes diagnosis.
Conclusion
C‐peptide declines over time in Wolfram syndrome and could potentially be used as a marker of diabetes progression in interventional studies for Wolfram syndrome, especially within the first 2 years after diabetes diagnosis.</abstract><cop>Former Munksgaard</cop><pub>John Wiley & Sons A/S</pub><pmid>34792267</pmid><doi>10.1111/pedi.13291</doi><tpages>7</tpages><oa>free_for_read</oa></addata></record> |
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| source | MEDLINE; Wiley Online Library All Journals |
| subjects | Adolescent Ambulatory Care Facilities - organization & administration Ambulatory Care Facilities - statistics & numerical data Chi-Square Distribution Child Children Clinical trials C‐peptide Diabetes Diabetes mellitus Diabetes Mellitus - epidemiology Diabetes Mellitus - etiology Diagnosis DIDMOAD Disease Progression Female Hb A1c Humans Longitudinal Studies Male neurodegenerative disease Neurodegenerative diseases Patients Peptides Washington - epidemiology Wolfram Syndrome - complications Wolfram Syndrome - epidemiology |
| title | Longitudinal progression of diabetes mellitus in Wolfram syndrome: The Washington University Wolfram Research Clinic experience |
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