%0 Journal Article
%T Diazoxide choline extended-release tablet in people with Prader-Willi syndrome: results from long-term open-label study.
%A Miller JL
%A Gevers E
%A Bridges N
%A Yanovski JA
%A Salehi P
%A Obrynba KS
%A Felner EI
%A Bird LM
%A Shoemaker AH
%A Angulo M
%A Butler MG
%A Stevenson D
%A Goldstone AP
%A Wilding J
%A Lah M
%A Shaikh MG
%A Littlejohn E
%A Abuzzahab MJ
%A Fleischman A
%A Hirano P
%A Yen K
%A Cowen NM
%A Bhatnagar A
%A  
%J Obesity (Silver Spring)
%V 32
%N 2
%D 2024 02 2
%M 37919617
%F 9.298
%R 10.1002/oby.23928
%X 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).<BR>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.<BR>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%).<BR>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.