Real-world treatment patterns & neurologist treatment satisfaction for Duchenne muscular dystrophy patients by ambulatory status in the United States


Clinical Management

Poster Number: T325


Jonathan Strober, MD, UCSF, Valeria Merla, MPH, Pfizer, Nate Posner, MPH, Pfizer, Inc., Anna Talaga, PhD, Pfizer, Ella Morton, BSc, Adelphi Real World, Bollington, UK, Halima Iqbal, BSc, Adelphi Real World, Bollington, UK, Emma Chatterton, MRes, Adelphi Real World, Bollington, UK, Niall Hatchell, MSc, Adelphi Real World, Bollington, UK

Introduction: Duchenne muscular dystrophy (DMD) is a genetic disorder associated with progressive muscle degeneration due to lack of dystrophin. Current treatment options can delay disease progression, but are non-curative, and many are largely mutation/exon specific. Here, we describe real-world treatment patterns for ambulatory and non-ambulatory DMD patients.

Methods: Data were drawn from the Adelphi DMD Disease Specific Programme™, a cross-sectional survey with retrospective data collection of physicians and their DMD patients conducted in the United States (US) from October 2022 – September 2023. Physicians completed online questionnaires for consecutive consulting DMD patients, reporting demographics, treatment history and satisfaction, and ambulatory status. Analyses were descriptive.

Results: Neurologists provided data on 235 male DMD patients. Mean [standard deviation; SD] patient age was 13.6 [7.3] years and 71% were ambulatory. Overall, 87% of ambulatory and 88% of non-ambulatory patients were prescribed treatment. Treatment was commonly mutation-specific medication (overall: 34%; ambulatory: 32%; non-ambulatory: 41%) or prednisone/prednisolone (overall: 29%). Mean [SD] time on a mutation-specific regimen was 3.2 [3.5] years (ambulatory: 2.5 [2.7] years; non-ambulatory: 4.6 [4.7] years). To manage symptoms, 41% of patients (ambulatory: 32%; non-ambulatory: 66%) were prescribed ≥1 additional medication (e.g., bisphosphonates). Side effects with current regimen were experienced by 72% of patients (ambulatory: 69%; non-ambulatory: 79%). Since treatment initiation, ambulation had “worsened”/“remained the same” for 34% of patients (ambulatory: 24%; non-ambulatory: 59%). Neurologists reported dissatisfaction/neutrality with current regimen for 25% of patients (ambulatory: 19%; non-ambulatory: 36%). Diminishing efficacy was the key neurologist-reported reason for lack of treatment satisfaction (40%).

Conclusion: One third of DMD patients did not achieve a delay in loss of ambulation despite prescribed treatment. Side effects were common and diminishing efficacy was a key reason for dissatisfaction with therapy. Combined with neurologist dissatisfaction, this highlights the need for long-term, well-tolerated treatment that can delay disease progression.