Diverse Trial Designs, Populations, and Outcomes: A Systematic Literature Review of Trials for the Treatment of Hereditary Angioedema Attacks.
Raffi Tachdjian, Nihal Narsipur, Paranjoy Saharia et al.
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In 30 seconds
This systematic literature review evaluated 12 phase 2-3 randomized clinical trials for on-demand treatments of hereditary angioedema (HAE) attacks. It found significant variability in trial designs and outcomes, with center-based trials showing longer treatment times (median, 215-630 min) compared to a home-based trial (35-51 min).
Key findings
- Center-based trials required treatment within 5-8 hours post-attack, while home-based treatment allowed immediate intervention.
- Median treatment times were 215-630 minutes for center-based trials versus 35-51 minutes for the home-based trial.
- Censoring rates in placebo arms exceeded 50% in some trials, affecting data comparability.
- Eight trials used time to onset of symptom relief as the primary endpoint, with varying definitions.
Why it matters
Understanding the heterogeneity in trial designs and outcomes is crucial for clinicians to interpret the efficacy of HAE treatments and to inform future research directions.