Last week, PPMD brought together leading experts to address steroids, stress dosing and the updated PJ Nicholoff Steroid Protocol. Drs. Aravindhan Veerapandiyan, David Weber, and Anne Marie Sbrocchi shared valuable insights on the management of steroids, importance of stress dosing, and helping families navigate the effects of long-term corticosteroid treatment with the PJ Nicholoff Steroid Protocol. 

First published ten years ago, the PJ Nicholoff Steroid Protocol is a critical document in the management of steroid side effects, adrenal suppression, and stress dosing in times of illness or injury. This new, updated version – produced in partnership with PPMD by the OPTIMIZE DMD Consortium, a group of physicians with expertise in Duchenne endocrine care – builds upon the original document with expanded guidance and recommendations for the management of steroid therapy. 

Dr. Veerapandiyan opened the discussion with an overview of steroid therapy in Duchenne, outlining how steroids work and their potential side effects. He compared prednisone, Emflaza (deflazacort) and Agamree (vamorolone) and the clinical considerations that healthcare providers and families should weigh when initiating and managing long-term steroid therapy.

Dr. Sbrocchi then explained the long-term effects of steroids and risk of adrenal suppression, emphasizing that emergency preparedness is crucial and potentially life-saving. She highlighted the importance of working with your care team to ensure you have an emergency plan in place and using the PJ Nicholoff Protocol can help guide your individual plan. 

Dr. Weber took a deep dive into the updated protocol, including 6 critical concepts:

1. All individuals (and their caregivers) taking steroids must recognize the signs & symptoms of adrenal insufficiency. 
2. Everyone taking steroids must have a stress steroid plan. 
3. Taking steroids for more than 2 weeks causes adrenal suppression. 
4. If stopping steroids, it is important to slowly taper the dose to reduce side effects. 
5. Continue to use stress steroids during a steroid taper until the adrenal glands have recovered. 
6. Be careful when changing from one steroid or steroid regimen to another. 

 

The updated protocol provides a roadmap for safe and effective steroid management, reflecting the current knowledge and best practices in Duchenne care.

By staying informed about these critical concepts and maintaining open communication with healthcare providers, families can navigate steroid therapy with greater confidence and be better prepared for any situation that may arise.

 

Resources

• Download the PJ Nicholoff Steroid Protocol here 
• Vamorolone Information Sheet for Families
• Discussing Vamorolone (Agamree) with your Healthcare Provider: What Every Person with Duchenne and their Family Must Know