Evidence map›Paper›PMID 39331535›Full record

ReviewClinical and translational allergy2024

Advent of oral medications for the treatment of hereditary angioedema.

Anna Valerieva, Teresa Caballero, Markus Magerl, Joao P Frade, Paul K Audhya, Timothy Craig

Abstract readReview
In one paragraph

Review in Clinical and translational allergy, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

9 citing papers in PubMed.

  1. Article
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors.

Anna ValerievaDepartment of Allergology, Medical University of Sofia, University Hospital "Alexandrovska", Sofia, Bulgaria.ORCID https://orcid.org/0000-0002-0417-9840
Teresa CaballeroServicio de Alergia, Hospital Universitario la Paz, IdiPAZ, CIBERER U754, Madrid, Spain.
Markus MagerlInstitute of Allergology IFA, Charité - Universitätsmedizin Berlin, Berlin, Germany.
Joao P FradeKalVista Pharmaceuticals, Cambridge, Massachusetts, USA.
Paul K AudhyaKalVista Pharmaceuticals, Cambridge, Massachusetts, USA.
Timothy CraigDepartments of Medicine, Pediatrics, and Biomedical Sciences, Penn State University, Hershey, Pennsylvania, USA.ORCID https://orcid.org/0000-0002-7774-4855

Funding

KalVista Pharmaceuticals
6 · The paper itself

Abstract

backgroundHereditary angioedema (HAE) is a rare genetic disorder characterized by unpredictable, debilitating episodes of submucosal and/or subcutaneous tissue swelling, which may be life-threatening depending on anatomic location. The two primary management strategies for HAE are ready access to effective on-demand treatment in all patients and the prevention of attacks (short-term prophylaxis [STP] and long-term prophylaxis [LTP]) in appropriate patients. All approved on-demand and most LTP medications require subcutaneous or intravenous administration. Injection-related challenges include trypanophobia (fear of needles), difficulty with self-administration, injection-site reactions (e.g., pain, erythema, bleeding, bruising), and anxiety-all contributing to poor compliance and administration delays. Oral HAE treatments may improve outcomes by reducing treatment barriers.

aimTo review oral therapies, approved or in development, for on-demand treatment and/or prevention of HAE attacks. MATERIALS AND

methodsTo provide a comprehensive review, data was obtained from publicly available resources through a targeted PubMed literature review and supplemented by information provided on company websites (search cutoff of May 31, 2024).

resultsBerotralstat, an oral plasma kallikrein (PKa) inhibitor, is approved for LTP. Sebetralstat, another PKa inhibitor, is the investigational first oral on-demand HAE treatment to complete a phase 3 trial. Deucrictibant, an oral bradykinin B2 receptor antagonist, has completed phase 2 trials for on-demand therapy and LTP. Several other oral PKa inhibitors (ATN249, VE-4666, and VE-4062) are in early development for LTP.

conclusionSubstantial advances have been made in the development of oral treatments for HAE. These treatments have the potential to improve and optimize clinical outcomes, satisfaction, and quality of life among patients with HAE.

Indexed as

berotralstatdeucrictibanthereditary angioedemaoralsebetralstat

Identifiers

PMID39331535
PMCPMC11431061

What OpenQuestion holds

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Registered trials

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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 OpenQuestion graph.