Corticosteroids Delay Need for Nocturnal Non-Invasive Ventilatory Support in Males with Duchenne Muscular Dystrophy

Topic:

Clinical Management

Poster Number: 14 S

Author(s):

Allyson Brown, MD, Oregon Health & Science University/Doernbecher Children's Hospital, Matthew Alexander, PhD, University of Alabama at Birmingham, AKM Fazlur Rahman, PhD, University of Alabama at Birmingham, Shelley Coskery, RN, MSN, CRNP, University of Alabama at Birmingham/Children's of Alabama, Ammar Saadoon Alishlash, MD, University of Alabama at Birmingham/Children's of Alabama, Michael Lopez, MD, PhD, University of Alabama at Birmingham/Children's of Alabama, Bradley Troxler, MD, University of Alabama at Birmingham/Children's of Alabama

Background
Duchenne muscular dystrophy (DMD) is the most common hereditary neuromuscular disease worldwide characterized by progressive muscle weakness, cardiomyopathy, and neuromuscular respiratory failure. This respiratory failure first presents as nocturnal hypoventilation requiring nocturnal non-invasive positive pressure ventilation (N-NIPPV) and emerges temporally with the loss of ambulation. While mainstay therapies like corticosteroids (CSs) have consistently been shown to delay progression of muscle weakness, their impact on respiratory outcomes remains poorly defined.
Objective
To determine if treatment with CSs delays the need for initiation of N-NIPPV in male patients with DMD and to elucidate if the duration of CS therapy influences this effect.
Methods
A retrospective chart review was conducted on all males with DMD followed at a tertiary pediatric center from 2018-2024. Data from 127 male DMD patients were categorized based on corticosteroid use. Age at first polysomnography recommending N-NIPPV was compared across steroid-naive and steroid-exposed groups based on length of CS exposure using Kaplan-Meier survival analysis and Cox regression.
Results
Compared to steroid-naïve patients, patients treated with CSs had a significantly delayed need for the initiation of N-NIPPV. This relationship was most notable after age 15 and was not affected by either the length of time on CSs or by race.
Conclusion
Treatment with CSs delays the onset of respiratory failure in the form of nocturnal hypoventilation requiring N-NIPPV in males with DMD. This impact is not driven by the length of time on CSs, supporting that even short courses of CSs provide clinical benefit in respiratory outcomes. As the effect is most evident after age 15, it also supports a clinical benefit to continuing CSs beyond loss of ambulation to optimize respiratory outcomes.