Diazoxide choline extended-release tablet in people with Prader-Willi syndrome: results from long-term open-label study
Author(s)
Type
Journal Article
Abstract
OBJECTIVE: 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). METHODS: 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. RESULTS: 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%). CONCLUSIONS: 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.
Date Issued
2024-02
Date Acceptance
2023-09-06
Citation
Obesity, 2024, 32 (2), pp.252-261
ISSN
1071-7323
Publisher
Wiley
Start Page
252
End Page
261
Journal / Book Title
Obesity
Volume
32
Issue
2
Copyright Statement
© 2023 The Authors. Obesity published by Wiley Periodicals LLC on behalf of The Obesity Society. This article has been contributed to by U.S. Government
employees and their work is in the public domain in the USA. This is an open access article under the terms of the Creative Commons Attribution-NonCommercial License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.
employees and their work is in the public domain in the USA. This is an open access article under the terms of the Creative Commons Attribution-NonCommercial License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.
License URL
Identifier
https://www.ncbi.nlm.nih.gov/pubmed/37919617
Publication Status
Published
Coverage Spatial
United States
Date Publish Online
2023-11-02