An expert review, written by Drs. Miller, Shoemaker, Salehi, and Corletto, offers practical clinical guidance for the use of diazoxide choline extended-release (DCCR/VYKAT™ XR), the first FDA-approved treatment for hyperphagia in Prader-Willi syndrome (PWS).
This review, published in the Journal of the Endocrine Society, incorporates findings from the DESTINY-PWS clinical trial program and the real-world clinical experience of VYKAT™ XR use in the PWS population so far, into actionable recommendations, including:
- Patient Selection: Who may benefit from DCCR treatment
- When to begin therapy based on hyperphagia progression
- Checklist to complete prior to initiating treatment
- Weight-based dosing and titration protocols
- Monitoring recommendations for blood glucose, edema, and cardiopulmonary risks
- Management of common side effects, including hyperglycemia and fluid retention
- Drug interaction considerations and dose-adjustment strategies
- Additional considerations and guidance for patients with pre-diabetes, diabetes, edema, obesity, sleep apnea, or other complex medical needs
Key Takeaways
The paper highlights several important considerations for clinicians, caregivers, and families as DCCR begins broader use in the PWS community:
- The first several months of treatment require close monitoring for hyperglycemia (high blood sugar) and edema
- Most side effects observed in clinical trials were manageable with careful monitoring and dose adjustments
- Optimizing management of conditions such as diabetes, edema, and sleep apnea before starting treatment may improve safety and tolerability
- Successful treatment requires close collaboration between caregivers and a multidisciplinary medical team
As DCCR enters broader clinical use, this publication provides one of the first detailed expert frameworks for real-world implementation. Ongoing clinical experience, data from the Global PWS Registry, and future studies will continue to refine best practices and expand the understanding of long-term treatment outcomes in PWS.
Read the full paper and share with your healthcare provider: Diazoxide choline extended-release (DCCR) use in Prader-Willi syndrome: patient selection, dosing, and management
