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.

View abstract

Authors

Iff J, Zhong Y, Tuttle E, Gupta D, Paul X, Henricson E