Glucocorticoid Use and Delayed Respiratory Decline in Duchenne Muscular Dystrophy Patients: A Real-World Analysis


Topic:

Other

Poster Number: 76 S

Author(s):

Steven Woods, PharmD, Catalyst Pharmaceuticals, Bridget McGowan, MD, Ann and Robert H. Lurie Children's Hospital of Chicago, Ashley Martin, PhD, BluePath Solutions, Alexa Gordon, MS, BluePath Solutions, Sana Mirza, MPH, BluePath Solutions, Paula Alvarez, MBA, MS, Rph, Critical Intelligence Consulting, Oscar Mayer, MD, The Children's Hospital of Philadelphia

Introduction: In Duchenne muscular dystrophy (DMD), glucocorticoids (GCs) can play a crucial role in preserving pulmonary function by slowing the progression of respiratory muscle weakness. Here, we examined whether long-term GC use was related to a reduced risk of respiratory complications, particularly during adolescence and early adulthood.

Methods: A retrospective cohort study used 2017–2022 Medicaid claims to assess respiratory outcomes among male DMD patients aged ≤30 years. Long-term GC users (≥90 days of use with ≤60-day gaps) were compared to non-users (no GC use during the study period and in the 12 months prior). Adjusted odds ratios (aOR) were estimated for discrete age groups [10, 15, 20, 25, 30 years] to examine the impact of long-term GC use for pneumonia, upper respiratory infections (URIs), and respiratory failure, adjusting for race/ethnicity, comorbidity burden, and follow-up duration.

Results: Among 2,273 male DMD patients (1,365 GC users; 908 non-GC), GC users had significantly lower odds of pneumonia at ages 10 and 15 (aOR = 0.64 and 0.70, respectively). Although a protective effect was suggested at age 20 (aOR = 0.76), it was not statistically significant. GC users also had reduced odds of respiratory failure at ages 15 and 20 (aOR = 0.78 and 0.75, respectively). All p-values were <0.05 for significant comparisons. No significant differences in URIs were observed between groups at any age. Conclusion: GC use was associated with lower odds of pneumonia and respiratory failure through mid-adolescence, with possible continued benefit into early adulthood. These age-stratified findings suggest that GCs delay respiratory progression in DMD.