Miller, Jennifer L, Gevers, Evelien, Bridges, Nicola 
ORCID: 0000-0002-2915-4877, Yanovski, Jack A ORCID: 0000-0001-8542-1637, Salehi, Parisa, Obrynba, Kathryn S, Felner, Eric I, Bird, Lynne M, Shoemaker, Ashley H, Angulo, Moris et al (show 14 more authors)
(2024)
Diazoxide choline extended-release tablet in people with Prader-Willi syndrome: results from long-term open-label study.
Obesity (Silver Spring, Md.), 32 (2).
pp. 252-261.
Abstract
<h4>Objective</h4>This study assessed the effect of 1-year administration of diazoxide choline extended-release tablet (DCCR) on hyperphagia and other complications of Prader-Willi syndrome (PWS).<h4>Methods</h4>The authors studied 125 participants with PWS, age ≥ 4 years, who were enrolled in the DESTINY PWS Phase 3 study and who received DCCR for up to 52 weeks in DESTINY PWS and/or its open-label extension. The primary efficacy endpoint was Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score. Other endpoints included behavioral assessments, body composition, hormonal measures, and safety.<h4>Results</h4>DCCR administration resulted in significant improvements in HQ-CT (mean [SE] -9.9 [0.77], p < 0.0001) and greater improvements in those with more severe baseline hyperphagia (HQ-CT > 22). Improvements were seen in aggression, anxiety, and compulsivity (all p < 0.0001). There were reductions in leptin, insulin, and insulin resistance, as well as a significant increase in adiponectin (all p < 0.004). Lean body mass was increased (p < 0.0001). Disease severity was reduced as assessed by clinician and caregiver (both p < 0.0001). Common treatment-emergent adverse events included hypertrichosis, peripheral edema, and hyperglycemia. Adverse events infrequently resulted in discontinuation (7.2%).<h4>Conclusions</h4>DCCR administration to people with PWS was well tolerated and associated with broad-ranging improvements in the syndrome. Sustained administration of DCCR has the potential to reduce disease severity and the burden of care for families.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | C601/C602 Investigators, Humans, Prader-Willi Syndrome, Hyperphagia, Diazoxide, Insulin, Body Composition, Child, Preschool |
| Divisions: | Faculty of Health and Life Sciences Faculty of Health and Life Sciences > Institute of Life Courses and Medical Sciences |
| Depositing User: | Symplectic Admin |
| Date Deposited: | 21 Mar 2024 14:55 |
| Last Modified: | 21 Mar 2024 14:55 |
| DOI: | 10.1002/oby.23928 |
| Open Access URL: | https://onlinelibrary.wiley.com/doi/10.1002/oby.23... |
| Related URLs: | |
| URI: | https://livrepository.liverpool.ac.uk/id/eprint/3179799 |
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