In addition to a wide range of case reports, reviews, and small case series, here are summaries of recently published HAE-related scientific papers. Data search was undertaken on 21 March 2025.
An evaluation of sebetralstat as the first oral on-demand therapy for hereditary angioedema
Henriette Farkas and Zsuzsanna Balla
The authors review sebetralstat, a promising option for rapid and effective on-demand treatment of HAE attacks. They conclude that oral sebetralstat provides a convenient alternative to injectable treatments, allowing patients to take the drug at the first sign of an attack, helping to resolve them quickly. They believe its chemical properties indicate it has potential for reliable control of HAE attacks.
(Expert Opinion on Pharmacotherapy, 20 March 2025
Impact of dental procedures on hereditary angioedema attacks: An exploratory observational study
Valentin Nadasan, et al
The authors evaluate whether patients with HAE receive appropriate treatment following an HAE attack associated with a dental procedure. In diagnosed patients, adequate HAE-specific medications were mostly used. However, a proportion of patients refrained from dental treatment or were denied treatment by professionals, due to fears of HAE attacks.
(Oral Health and Preventive Dentistry, 14 March 2025)
Hereditary angioedema with normal C1 inhibitor: An updated international consensus paper on diagnosis, pathophysiology and treatment
Bruce L Zuraw, et al
The authors propose recommendations for the management of HAE with normal C1-inhibitor (HAE-nC1INH). They suggest their review and expert opinion on best practice will support physicians to better manage patients with HAE-nC1INH.
(Clinical Reviews in Allergy and Immunology, 7 March 2025)
HAE International (HAEi) and the US Hereditary Angioedema Association (HAEA) supported this important work on HAE with normal C1 inhibitor, and we’ll bring you the inside scoop on this project and publication in the next edition of Global Perspectives.
Hereditary angioedema (HAE) in China: Advancing awareness, access, advocacy and alliances from the greater bay area to the global HAE community
Philip H Li, et al
The authors provided an overview of the progress made in managing HAE in China. By bridging the gap between east and west, a Greater Bay Area HAE Alliance is providing optimal patient care. The authors conclude that uniting as a worldwide HAE community will significantly advance efforts to improve care for those affected by HAE.
(Clinical and Experimental Allergy, 4 March 2025)
Hereditary angioedema with normal C1 inhibitor: A quarter century of forward progress and persisting obstacles
Sandra C Christiansen, et al
The authors attempt to bring the numerous scientific developments over the past 25 years into a proposed classification scheme to help decision-making when doctors evaluate patients with recurrent angioedema. The authors hope that this approach will lay the groundwork for future advances in our understanding of HAE-nl-C1INH while bringing patients ever closer to the goal of leading a normal life.
(Journal of Allergy and Clinical Immunology – In Practice, in press 6 March)
Interplay between on-demand treatment trials for hereditary angioedema and treatment guidelines
Danny M Cohn, et al
The authors note that over the last 20 years, guidelines for HAE have undergone substantial revision. The paper reviews the evolution of on-demand treatment guidelines, the trials and data that supported revisions, and the changes to trial designs necessary to comply with guidelines.
(Journal of Allergy and Clinical Immunology, March 2025)
Clinical response and corresponding blood transcriptome pathways before and after treatment of hereditary angioedema prodromes compared to active swelling attacks
Debajyoti Ghosh, et al
The authors investigated whether there was value in treating HAE type-1 patients during prodromal symptoms. They conclude from their research that in patients who have a well-defined prodrome that historically leads to an attack, treatment at the prodrome stage is justified.
(J Allergy Clin Immunol, March 2025)
A COSMIN systematic review of instruments for evaluating health-related quality of life in people with hereditary angioedema
Irene Baroni, et al
The authors looked at the tools currently used to evaluate quality of life in people with HAE. They conclude that two, HAE-QoL and AE-QoL have evidence to support their use, but they believe more data is needed on their suitability amongst younger patients and disease specific elements in them. They believe doing so will improve the accuracy of patient-reported outcome measures in HAE.
(Health and Quality of Life Outcomes, February 2025)
Correction: Uncovering a novel SERPING1 pathogenic variant: Insights into the aggregation of C1-INH in hereditary angioedema
Lingxi Jiang, et al
The authors corrected an earlier paper that suggested they had identified a new genetic variant in HAE. They note that this variant was described by experts in 2022. (The article being corrected was published in September 2024.)
(Orphanet Journal of Rare Diseases, February 2025)
Indirect treatment comparison of lanadelumab and a C1-esterase inhibitor in pediatric patients with hereditary angioedema
Maureen Watt, et al
The authors set out to compare the efficacy and safety of lanadelumab versus other approved long-term prophylaxis treatments in children with HAE. Reviewing two papers that indirectly compared treatment, the authors found a trend toward greater efficacy and fewer adverse events in patients treated with lanadelumab every two weeks. They suggest further studies of larger groups of patients could assess this further.
(Journal of Comparative Effectiveness Research, February 2025)
Real-world effectiveness of lanadelumab in hereditary angioedema: Multicountry INTEGRATED observational study
Markus Magerl, et al
The authors aimed to find out the effectiveness of lanadelumab on attack-free rate. They did this by looking back at the medical records of patients with HAE who were treated with this medicine. The authors conclude that long-term prophylaxis with lanadelumab was effective in improving the attack-free rate in patients with HAE, and that effectiveness was rapid, starting from the first month of beginning treatment.
(Journal of Allergy and Clinical Immunology: In Practice, February 2025)
Worldwide prevalence of hereditary angioedema: A systematic review and meta-analysis
Samuel A Fisch, et al
The authors indicate that the true prevalence of HAE is uncertain. Through their research, they indicate that the available studies suggest the global prevalence of HAE is 1-2 individuals per 100,000 people. They believe a true estimate of prevalence will inform care for HAE.
(International Archives of Allergy and Immunology, January 2025)
New drugs for the treatment of hereditary angioedema
Giulia Costanzo, et al
The authors wanted to review existing and future treatment options for HAE. It is their expert opinion that innovative therapies may allow individualized action plans and reduce the complexity of treatment. They conclude that long-term prophylaxis with longer administration intervals and oral on-demand therapies will have a key role in the future.
(Expert Opinion in Biological Therapy, January 2025)
Efficacy and safety of donidalorsen in hereditary angioedema with C1 inhibitor deficiency: A systematic review and a meta analysis
Adarsh Raja, et al
The authors consider data currently available on a potential new treatment for HAE, called donidalorsen. They suggest that it significantly reduced the frequency of attacks, with dosing every four weeks being superior to every eight weeks. They also suggest the rate of side effects was the same for people treated with donidalorsen and people treated with placebo. The authors conclude that more frequent dosing may optimize treatment outcomes with donidalorsen in HAE.
(Archives of Dermatological Research, December 2024)
Advances in the pathogenesis of hereditary angioedema
Xiang-Yi Cui and Yu-Xiang Zhi
This review summarizes the recent progress in classifying and understanding the underlying processes that cause HAE, and the role this may have in leading to better diagnosis and treatment of the disease.
(Zhongguo Yi Xue Ke Xue Yuan Xue Bao, December 2024)
Hereditary angioedema in children: Review and practical perspective for clinical management
A Pagnier, et al
The authors review all aspects of how HAE is diagnosed and managed in children. They comment that lanadelumab (given subcutaneously) and berotralstat (given orally) have made long-term prophylaxis more usable in children with HAE. They conclude that management of HAE is particularly challenging in children and requires multiple stakeholders.
(Pediatric Allergy and Immunology, December 2024)