SynthesisAdvances in therapy2026
Diverse Trial Designs, Populations, and Outcomes: A Systematic Literature Review of Trials for the Treatment of Hereditary Angioedema Attacks.
Synthesis in Advances in therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
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Authors and funding
9 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionHereditary angioedema (HAE) is a rare genetic disorder affecting approximately 1 in 50,000 people. As of July 2025, five on-demand treatments for HAE attacks have been approved based on phase 2/3 and phase 3 randomized clinical trials (RCTs). This systematic literature review (SLR) aimed to evaluate clinical trial designs, populations, and outcomes to better understand factors influencing observed treatment effects.
methodsFollowing health technology assessment methodology and standards, a comprehensive SLR was conducted through October 2024 using MEDLINE, MEDLINE In-Process, Embase, and the Cochrane Library. Eligible studies were phase 2-3 RCTs evaluating ecallantide, icatibant, plasma-derived C1 esterase inhibitor (C1-INH), recombinant human C1-INH (rhC1-INH), or sebetralstat, and reporting efficacy (e.g., time to onset of symptom relief [TOSR] or time to complete resolution [TTCR]). Assessments included trial design, attack characteristics, and patient-reported outcome (PRO) measures.
resultsOf the 12 RCTs evaluated, 11 were center-based and 1 was home-based with e-diary collection. Center-based trials required presentation within 5-8 h after attack onset, whereas the home-based trial instructed immediate treatment, resulting in faster treatment times (median, 215-630 min vs. 35-51 min, respectively). Baseline attack severity was a key differentiator between trials, with some trials requiring a minimum severity of ≥ 30 mm or ≥ 50 mm on the visual analog scale. Censoring criteria differed in timing and conditions (e.g., rescue medication, redosing), with placebo arm censoring rates exceeding 50% in some trials. Eight trials used TOSR as the primary endpoint, measured by six distinct PRO instruments with variable definitions of improvement and success.
conclusionSubstantial heterogeneity in trial design, attack eligibility criteria, redosing and rescue protocols, censoring rules, and endpoint definitions limit cross-trial comparability of on-demand HAE therapies. Future comparative efficacy research should prioritize harmonization of endpoint definitions, map across PRO instruments, and adjust for treatment effect modifiers.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.