Real-world evidence of eteplirsen treatment effects in patients with Duchenne muscular dystrophy in the USA
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, an...
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| Main Authors: | , , , , , |
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| Format: | Book |
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Becaris Publishing Limited,
2023-08-01T00:00:00Z.
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| Online Access: | Connect to this object online. |
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| Summary: | 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. |
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| Item Description: | 10.57264/cer-2023-0086 2042-6313 |