TTE
10 evidence topics · 1 source
Objective
Mortality outcome
Location and study date
U.S. claims observation period
Study design
Underlying data design
Eligibility criteria
Participant sex criterion
Treatment
Observed treatment strategies
| Strategy | Quoted group label |
|---|---|
| Exon-skipping therapy plus glucocorticoids | “PMO + GCs” |
| Glucocorticoids without a PMO | “GCs-only” |
Study outcomes
Rationale for using All-Cause mortality as a clinical endpoint
Not applicable.
Statistical analysis description
Number of participants (Planned and analyzed)
Description of analysis sets
Confounding and censoring adjustment
Results
Participant disposition
Baseline characteristics
Efficacy results
Eteplirsen, casimersen, and golodirsen survival analysis: 48 pooled emulated trials
Eteplirsen, casimersen, and golodirsen survival analysis: 66 pooled emulated trials
Health-related quality of life and patient-reported outcomes
No evidence found.
Safety results
No evidence found.
Study limitations
Summary
This retrospective claims analysis pooled eteplirsen, casimersen, and golodirsen, rather than evaluating z-rostudirsen. Weighting addressed measured treatment-selection and censoring factors but does not make the comparison randomized. The 48-trial pooled estimate was HR 0.303 (95% CI 0.119–0.769), whereas the 66-trial estimate was HR 0.477 (95% CI 0.194–1.169). The latter interval includes 1. Results therefore depend on the pooled analysis and do not establish agent-specific effects.