Evidence map›Paper›PMID 41582175›Full record

ArticleAllergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology2026

Attack rate reductions following berotralstat initiation among US patients with hereditary angioedema in the real-world.

Mark Davis-Lorton, Raffi Tachdjian, Lorena Lopez-Gonzalez, Sean D MacKnight, Ramya Ramasubramanian, François Laliberté, Patrick Gillard, Meri LiVecchi, Sandra Nestler-Parr, William R Lumry

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Article in Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology, 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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1 · What the graph read from it

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4 · The record

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5 · Who and what money

Authors and funding

10 authors.

Mark Davis-LortonENT and Allergy Associates, Tarrytown, NY, USA.
Raffi TachdjianDepartment of Pediatrics, University of California Los Angeles, 1301 20th Street, Suite 380, Los Angeles, Santa Monica, CA, 90404, USA. ucladoc@gmail.com.
Lorena Lopez-GonzalezBioCryst Pharmaceuticals, Inc., Durham, NC, USA.
Sean D MacKnightGroupe d'analyse SRI, Montreal, QC, Canada.
Ramya RamasubramanianAnalysis Group, Inc., Los Angeles, CA, USA.
François LalibertéGroupe d'analyse SRI, Montreal, QC, Canada.
Patrick GillardBioCryst Pharmaceuticals, Inc., Durham, NC, USA.
Meri LiVecchiBioCryst Pharmaceuticals, Inc., Durham, NC, USA.
Sandra Nestler-ParrBioCryst Pharmaceuticals, Inc., Durham, NC, USA.
William R LumryAllergy and Asthma Research Associates, Dallas, TX, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundHereditary angioedema (HAE) causes recurring swelling attacks, leading to substantial disease burden. This real-world, retrospective study aimed to evaluate HAE attack rates before and after berotralstat initiation stratified by patients' baseline attack frequency.

methodsSpecialty Pharmacy data from Optime Care, Inc. (12/2020-01/2024), the sole berotralstat dispenser in the United States, were analyzed. Eligible patients had  ≥ 2 berotralstat dispensings (first =  index) and ≥ 1 self-assessment of attacks at baseline (90-days pre-index) and follow-up (first-to-last dispensing). Patients were classified by HAE type (based on laboratory measurements) and baseline attacks (≥ 5, 2-4, 1, and 0 attacks/month). Follow-up attack rates were compared with baseline using mean differences, confidence intervals, and P-values. Among those with 0 baseline attacks/month, proportions with 0 follow-up attacks/month were assessed.

resultsOf 390 eligible patients with HAE with C1 esterase inhibitor (C1INH) deficiency (HAE-C1INH) and 311 with HAE with normal C1INH (HAE-nC1INH), most were female (64.1% and 77.5%) with mean ages of 39.3 and 48.1 years, respectively. Mean attack rates decreased from 2.50 to 0.79 attacks/month (HAE-C1INH) and from 4.59 to 1.68 attacks/month (HAE-nC1INH) at 12-months of berotralstat treatment (both P < 0 .001), with sustained reductions at 18-months. Patients with ≥ 1 baseline attack/month experienced significantly lower attack rates after berotralstat initiation. Among patients with 0 baseline attacks/month, most also maintained 0 attacks/month in each follow-up interval (HAE-C1INH: 70-85%; HAE-nC1INH: 61-81%).

conclusionBerotralstat was associated with significant and sustained reductions in attack rates among patients with HAE, regardless of baseline attack rate. Patients with 0 baseline attacks/month maintained low attack rates following berotralstat initiation.

Indexed as

BerotralstatHereditary angioedemaLong-term prophylaxisRare diseaseReal-world evidence

Identifiers

PMID41582175
PMCPMC13007376

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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.