Real-world evidence of eteplirsen treatment effects in patients with Duchenne muscular dystrophy in the USA
Journal of Comparative Effectiveness Research, 2023
Aim
To evaluate treatment effects of eteplirsen among patients with Duchenne muscular dystrophy.
Methods
Using real-world claims and electronic medical record data, this retrospective comparative analysis assessed eteplirsen-treated and control cohorts matched by age, disease progression state, and pre-index period healthcare resource utilization. Poisson regression was used to evaluate eteplirsen effects on healthcare resource utilization outcomes.
Results
Eteplirsen was associated with statistically significant reductions in rates of hospital encounters (31%), emergency room visits (31%), need for pulmonary management (33%), cardiac management (21%), tracheostomy (86%), and assisted ventilation (39%) versus the control group. Other assessed outcomes favored eteplirsen numerically but did not all reach statistical significance.
Conclusion
Eteplirsen-treated patients had reduced rates of multiple healthcare resource utilization measures versus matched controls.