22-ANGR-120
10 evidence topics · 3 sources
Objective
Survival outcome evaluated
Location and study date
Study design
Treated-cohort data source
Eligibility criteria
Treated study population
Treatment
Observed treatment
Study outcomes
Rationale for using overall survival as a clinical endpoint
Not applicable.
Statistical analysis description
Number of participants (Planned and analyzed)
Description of analysis sets
External-control construction
Results
Participant disposition
Treated-cohort disposition
| Status | Quoted result |
|---|---|
| Died, n (%) | “29 (5.0)” |
| Censored, n (%) | “550 (95.0)” |
Baseline characteristics
Age at treatment initiation
| Characteristic | Quoted result |
|---|---|
| Age, years, mean and standard deviation | “11.9 ± 6.4” |
Efficacy results
Eteplirsen versus natural-history survival association
| Measure | Quoted result |
|---|---|
| Median survival age with eteplirsen, years | “32.8” |
| Median survival age in natural-history cohorts, years | “27.4” |
| Hazard ratio | “0.34” |
| 95% confidence interval | “0.23–0.50” |
Health-related quality of life and patient-reported outcomes
No evidence found.
Safety results
No evidence found.
Study limitations
Summary
The study compared administrative data for 579 eteplirsen-treated participants with reconstructed patient-level information for 1,224 natural-history controls. It was not a randomized comparison. Consequently, the reported survival association cannot establish an unbiased causal treatment effect or a comparison with z-rostudirsen. Reconstruction of external controls and differences between data sources limit comparability.