Nonprofit releases updated PJ Nicholoff Steroid Protocol for DMD
Guidance intended to aid healthcare providers in managing corticosteroid use
Parent Project Muscular Dystrophy (PPMD), a nonprofit dedicated to ending Duchenne muscular dystrophy (DMD), has published an updated edition of the PJ Nicholoff Steroid Protocol, marking the 10th anniversary of its first release.
Called PJ’s Protocol, the guidance is designed to help healthcare providers manage corticosteroid use in DMD, particularly during times of physical stress such as injury or surgery.
Developed in collaboration with Optimize DMD, a global consortium working toward better care, education, and research in endocrine and bone health for people with Duchenne, the revised edition integrates a decade of progress in clinical research, the emergence of newly approved therapies, along with safety measures to improve patient outcomes.
“At PPMD, we are committed to ensuring that every individual with Duchenne receives the highest standard of care,” Rachel Schrader, PPMD’s vice president of clinical care and education, said in a press release. “This significant update to the PJ Nicholoff Steroid Protocol reflects the rapidly changing landscape of approved therapies and subsequent changes to clinical care. The protocol provides expert recommendations to better equip healthcare professionals and caregivers with the most up-to-date knowledge to safely manage patients experiencing illness, injury, or safely navigate other substantive changes to their steroid regimen.”
Guiding steroid use in DMD
Steroids, such as prednisone, Emflaza (deflazacort), and Agamree (vamorolone) are widely used in DMD to help preserve muscle function and ease inflammation. Long-term use is linked to an increased risk of adrenal insufficiency, when the body loses the ability to produce enough of the hormone cortisol, however.
During periods of physical stress, such as illness, injury, or surgery, the body requires higher cortisol levels to maintain critical functions. For people with adrenal insufficiency, failure to receive appropriate treatment can lead to an adrenal crisis, which is marked by severe fatigue, low blood pressure, vomiting, and, in extreme cases, shock.
The PJ Nicholoff Steroid Protocol is named after Phillip James Nicholoff, who lived with DMD and died at age 31 after surgery for bone fractures. A review of his medical records found he didn’t receive appropriate stress doses of steroids during his hospitalization and had complications after surgery that included respiratory distress and low blood pressure.
The protocol seeks to standardize emergency steroid management by providing healthcare providers with evidence-based guidance to recognize early signs of acute adrenal insufficiency, administer timely stress-dose steroids, and safely taper or reduce corticosteroids to prevent withdrawal complications.
The revised 2025 protocol includes six critical concepts. They are:
- the risk of adrenal insufficiency of individuals on chronic steroid therapy
- the challenging detection of adrenal insufficiency
- the importance of stress dosing for individuals under moderate or severe physiologic stress
- how to safely taper when decreasing or discontinuing steroids
- the role of stress-dose steroids at times of physiologic stress in those tapering off corticosteroids or on maintenance doses
- the extra care needed to lower the risk of adrenal insufficiency when transitioning from one steroid to another.
“The PJ Nicholoff Protocol is a ‘how-to’ guide designed to assist healthcare professionals in the prevention of adrenal insufficiency for people with Duchenne, a potentially serious complication of corticosteroid therapy,” said Leanne Ward, MD, a pediatric endocrinologist at the Children’s Hospital of Eastern Ontario and founder and chairwoman of the Optimize DMD consortium.
“The 2025 edition, developed under the expert leadership of Dr. David Weber (Children’s Hospital of Philadelphia) and Dr. Anne Marie Sbrocchi (Montreal Children’s Hospital), provides important updates, including the management of adrenal insufficiency for individuals receiving the novel steroid [Agamree],” Ward said. “On behalf of the Optimize DMD consortium, we are so grateful to the Nicholoff family for their enduring support toward international efforts aimed at the prevention of adrenal insufficiency in Duchenne muscular dystrophy.”
To support the rollout of the new protocol, PPMD will host a live webinar May 30 at 1 p.m. ET. The event will feature leading experts who will offer an overview of the revision and discuss its practical implementation in clinical practice.
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