Supplementary data: Real-world evidence of eteplirsen treatment effects in patients with Duchenne muscular dystrophy in the USA

<b>These are peer-reviewed supplementary data for the article '</b><b>Real-world evidence of eteplirsen </b><b>treatment effects in patients with </b><b>Duchenne muscular dystrophy in the USA</b><b>' published in the</b><b> </b><b><i>Journal of Comparative Effectiveness Research</i></b><b>.</b><b>Supplementary </b><b>Table 1: </b>Poisson Regression Analysis Results<b>Supplementary </b><b>Table 2:</b> Sensitivity Analyses Poisson Regressions in the Matched Treated and SoC Patients<b>Aim:</b> To evaluate treatment effects of eteplirsen among patients with Duchenne muscular dystrophy. <b>Methods:</b> 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. <b>Results:</b> 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. <b>Conclusion: </b>Eteplirsen-treated patients had reduced rates of multiple healthcare resource utilization measures versus matched controls.