| Title: |
Diazoxide choline extended‐release tablet in people with Prader‐Willi syndrome: results from long‐term open‐label study |
| Authors: |
Miller, Jennifer L.; Gevers, Evelien; Bridges, Nicola; Yanovski, Jack A.; Salehi, Parisa; Obrynba, Kathryn S.; Felner, Eric I.; Bird, Lynne M.; Shoemaker, Ashley H.; Angulo, Moris; Butler, Merlin G.; Stevenson, David; Goldstone, Anthony P.; Wilding, John; Lah, Melissa; Shaikh, M. Guftar; Littlejohn, Elizabeth; Abuzzahab, M. Jennifer; Fleischman, Amy; Hirano, Patricia; Yen, Kristen; Cowen, Neil M.; Bhatnagar, Anish |
| Publisher Information: |
Wiley |
| Publication Year: |
2024 |
| Collection: |
University of Glasgow: Enlighten - Publications |
| Description: |
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. |
| Document Type: |
article in journal/newspaper |
| File Description: |
text |
| Language: |
English |
| Relation: |
https://eprints.gla.ac.uk/313384/1/313384.pdf; Miller, J. L. et al. (2024) Diazoxide choline extended‐release tablet in people with Prader‐Willi syndrome: results from long‐term open‐label study. Obesity , 32(2), pp. 252-261. (doi:10.1002/oby.23928 ) (PMID:37919617) |
| DOI: |
10.1002/oby.23928 |
| Availability: |
https://eprints.gla.ac.uk/313384/; https://eprints.gla.ac.uk/313384/1/313384.pdf; https://doi.org/10.1002/oby.23928 |
| Rights: |
cc_by_nc_4 |
| Accession Number: |
edsbas.AB8F9F62 |
| Database: |
BASE |