To evaluate treatment effects of eteplirsen among patients with Duchenne muscular dystrophy. 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. 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. Eteplirsen-treated patients had reduced rates of multiple healthcare resource utilization measures versus matched controls.
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| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC10690424 | PMC |
| http://dx.doi.org/10.57264/cer-2023-0086 | DOI Listing |
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