Steroid Transitions: Clinical Decision-Making Across the DMD Disease Course

This episode examines how clinicians approach switching between corticosteroids and individualize steroid management over time. Dr. Chrzanowski notes increasing family interest in vamoralone, and that he presents available efficacy and safety data transparently—emphasizing non-inferiority on functional outcomes alongside a more favorable side effect profile. He finds vamoralone most beneficial in younger, still-growing patients; in post-pubertal individuals with limited growth potential, the height-related advantage diminishes.

The panel discusses the complexity of switching regimens, especially for non-ambulatory patients transitioning from low-dose or alternate-schedule corticosteroids to full-dose vamoralone at 6 mg/kg—a substantially higher steroid exposure. Some patients experience symptoms suggestive of adrenal suppression during this switch, including excessive fatigue and muscle aching. This has prompted many centers to implement a hydrocortisone taper to bridge the transition, though standardized protocols are still emerging.

Dr. Jayaraman reflects on how steroid management evolves with the patient. Older patients may prefer weekend dosing for tolerability. She reminds families that despite all the newer treatments, population-level data still support approximately three additional years of ambulation with steroids, as well as improved cardiac and pulmonary function and reduced scoliosis surgery rates.

The episode also addresses decision-making around steroid dose reduction in the non-ambulatory phase. Several clinical signals may prompt reassessment: bone health deterioration, cataract formation, weight gain impairing respiratory function, and caregiver burden. Dr. Jayaraman shares a case of a single mother of two boys with DMD who must lift a non-ambulatory child up flights of stairs—a situation in which daily steroids may not be the most appropriate recommendation. The panel reinforces that steroid management must be individualized to the patient's trajectory, family circumstances, and therapeutic context.

In the next episode, "DMD Treatment Algorithms: Initiating and Sequencing Therapies in New Patients," the panel walks through their clinical decision-making frameworks when introducing therapies to newly diagnosed patients.