Real-world evidence of eteplirsen treatment effects in patients with Duchenne muscular dystrophy in the USA
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.
Plain language summary
How eteplirsen treatment impacts the health of people living with Duchenne muscular dystrophy in the United States
What is this article about?
Duchenne muscular dystrophy (DMD) is a rare genetic disease. People with DMD do not make a protein called dystrophin. This leads to damage to all muscles, including the heart and the muscles used for breathing. Patients also lose the ability to walk and take care of themselves. Treatment for DMD is complex, and as the disease progresses, patients use more healthcare resources. Eteplirsen is a treatment for people with DMD, which is caused by mutations in a specific part of the gene, and helps produce shortened but functional dystrophin. Researchers looked at information from insurance claims and electronic medical records to compare healthcare resource use in people who received eteplirsen (n = 579) and those who received standard of care (n = 1296).
What were the results?
Patients receiving eteplirsen had significantly fewer hospital admissions (31%) and emergency room visits (31%). Patients also had a reduced need for special care of the lungs (pulmonary management) (33%), care for the heart (cardiac management) (21%), special surgery to access the windpipe (tracheostomies) (86%), and help with breathing (assisted ventilation) (39%) compared with patients who received standard of care.
What do the results mean?
These results suggest that people who received eteplirsen in routine clinical care settings used healthcare resources less often compared with patients who did not receive eteplirsen. Similar findings were seen in people receiving eteplirsen in clinical trials. These results suggest that eteplirsen treatment delays muscle damage in people with DMD.