{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,16]],"date-time":"2026-10-16T15:38:17Z","timestamp":1792165097647,"version":"4.4.0"},"reference-count":20,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2023,11,2]],"date-time":"2023-11-02T00:00:00Z","timestamp":1698883200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Obesity"],"published-print":{"date-parts":[[2024,2]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>This study assessed the effect of 1\u2010year administration of diazoxide choline extended\u2010release tablet (DCCR) on hyperphagia and other complications of Prader\u2010Willi syndrome (PWS).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>The authors studied 125 participants with PWS, age\u2009\u2265\u20094\u2009years, who were enrolled in the DESTINY PWS Phase 3 study and who received DCCR for up to 52\u2009weeks in DESTINY PWS and\/or its open\u2010label extension. The primary efficacy endpoint was Hyperphagia Questionnaire for Clinical Trials (HQ\u2010CT) score. Other endpoints included behavioral assessments, body composition, hormonal measures, and safety.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>DCCR administration resulted in significant improvements in HQ\u2010CT (mean [SE] \u22129.9 [0.77], <jats:italic>p<\/jats:italic>\u2009&lt;\u20090.0001) and greater improvements in those with more severe baseline hyperphagia (HQ\u2010CT\u2009&gt;\u200922). Improvements were seen in aggression, anxiety, and compulsivity (all <jats:italic>p<\/jats:italic>\u2009&lt;\u20090.0001). There were reductions in leptin, insulin, and insulin resistance, as well as a significant increase in adiponectin (all <jats:italic>p<\/jats:italic>\u2009&lt;\u20090.004). Lean body mass was increased (<jats:italic>p<\/jats:italic>\u2009&lt;\u20090.0001). Disease severity was reduced as assessed by clinician and caregiver (both <jats:italic>p<\/jats:italic>\u2009&lt;\u20090.0001). Common treatment\u2010emergent adverse events included hypertrichosis, peripheral edema, and hyperglycemia. Adverse events infrequently resulted in discontinuation (7.2%).<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>DCCR administration to people with PWS was well tolerated and associated with broad\u2010ranging improvements in the syndrome. Sustained administration of DCCR has the potential to reduce disease severity and the burden of care for families.<\/jats:p><\/jats:sec>","DOI":"10.1002\/oby.23928","type":"journal-article","created":{"date-parts":[[2023,11,3]],"date-time":"2023-11-03T04:06:27Z","timestamp":1698984387000},"page":"252-261","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":27,"title":["Diazoxide choline extended\u2010release tablet in people with <scp>Prader\u2010Willi<\/scp> syndrome: results from long\u2010term open\u2010label study"],"prefix":"10.1002","volume":"32","author":[{"given":"Jennifer L.","family":"Miller","sequence":"first","affiliation":[{"name":"Department of Pediatrics University of Florida College of Medicine  Gainesville Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Evelien","family":"Gevers","sequence":"additional","affiliation":[{"name":"Queen Mary University of London, Barts Health NHS Trust\u2013Royal London Children's Hospital  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2915-4877","authenticated-orcid":false,"given":"Nicola","family":"Bridges","sequence":"additional","affiliation":[{"name":"Chelsea and Westminster Hospital  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8542-1637","authenticated-orcid":false,"given":"Jack A.","family":"Yanovski","sequence":"additional","affiliation":[{"name":"Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health  Bethesda Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Parisa","family":"Salehi","sequence":"additional","affiliation":[{"name":"Seattle Children's Hospital  Seattle Washington USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathryn S.","family":"Obrynba","sequence":"additional","affiliation":[{"name":"Nationwide Children's Hospital  Columbus Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eric I.","family":"Felner","sequence":"additional","affiliation":[{"name":"Emory University School of Medicine  Atlanta Georgia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lynne M.","family":"Bird","sequence":"additional","affiliation":[{"name":"University of California, San Diego\/Rady Children's Hospital  San Diego California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ashley H.","family":"Shoemaker","sequence":"additional","affiliation":[{"name":"Vanderbilt University Medical Center  Nashville Tennessee USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Moris","family":"Angulo","sequence":"additional","affiliation":[{"name":"New York University Langone Health  Mineola New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Merlin G.","family":"Butler","sequence":"additional","affiliation":[{"name":"University of Kansas Medical Center  Kansas City Kansas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David","family":"Stevenson","sequence":"additional","affiliation":[{"name":"Stanford University  Palo Alto California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anthony P.","family":"Goldstone","sequence":"additional","affiliation":[{"name":"Department of Endocrinology, Imperial College Healthcare NHS Trust Hammersmith Hospital  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John","family":"Wilding","sequence":"additional","affiliation":[{"name":"University of Liverpool, Clinical Sciences Centre Aintree University Hospital  Liverpool UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melissa","family":"Lah","sequence":"additional","affiliation":[{"name":"Indiana University School of Medicine  Indianapolis Indiana USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M. Guftar","family":"Shaikh","sequence":"additional","affiliation":[{"name":"Royal Hospital for Children University of Glasgow  Glasgow UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elizabeth","family":"Littlejohn","sequence":"additional","affiliation":[{"name":"Sparrow Clinical Research Institute  Lansing Michigan USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M. 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