In addition to a wide range of case reports and small series, here are summaries of recently published HAE-related scientific papers. Data search undertaken 24 November 2024.
CRISPR-based therapy for hereditary angioedema
Danny M Cohn, et al
The authors report the results of a clinical trial of a prospective gene therapy for HAE. The authors state that a single dose of the medicine, code-named NTLA-2002, may provide lifelong control of angioedema attacks. Following the small-scale study, the authors conclude that the therapy reduced angioedema attacks and led to sustained reductions in the kallikrein levels in patients with HAE. They recommend that a larger trial be conducted.
(New England Journal of Medicine, 24 October 2024)
Hereditary angioedema (HAE) in children and adolescents: New treatment options
Maria Fasshauer and Bettina Wedi
The authors review the current treatment options for HAE type I and type II in children and adolescents. They also discuss developments and future therapeutic options.
(Allergologie select, 30 October 2024)
Unveiling the complexities of hereditary angioedema
Cristina Violeta Tutunaru, et al
The authors review the intricacies and diverse presentations of HAE, and the emerging biomarkers and innovations in the management and prevention of HAE.
(Biomolecules, 14 October 2024)
Dietary and physical trigger factors in hereditary angioedema: Self-conducted investigation and literature overview
Julia Zarnowski and Regina Treudler
The authors aimed to investigate the influence of nutrition and physical activity in people with HAE at a Leipzig ACARE center. Thirty patients completed a questionnaire about their life and lifestyle, which, the authors conclude, indicated that dietary factors and physical activity frequently led to an aggravation of HAE. The authors believe patients should be counseled about this when discussing trigger avoidance.
(Allergologie select, 14 November 2024)
Hereditary angioedema and venous thromboembolism: Where there’s smoke, there’s fire
Stephen P Grover
The author highlights and reviews recent epidemiological evidence linking HAE with a significantly increased risk of blood clots in veins (venous thromboembolism or VTE). The review also discusses the impact of HAE-specific medicines on VTE and blood clotting.
(Seminars in Thrombosis and Hemostasis, 17 October 2024)
Hereditary angioedema due to C1-inhibitor deficiency: current therapeutic approaches
Giulia Costanzo, et al
The authors review currently approved drugs for HAE and how treatments are developed. The authors conclude that the availability of newer therapeutic options has led to more individualized treatment plans for patients. The opportunity to switch from one treatment to another has also become easier. The authors foresee attack-free remission becoming a future possibility.
(Current Opinion in Allergy and Clinical Immunology, December 2024)
Structural basis for the inhibition of βFXIIa by garadacimab
Ieva Drulyte, et al
The authors examine both activated factor XII (FXIIa) and garadacimab using an electron microscope, to better understand how they interact and how the chemical structure of both can provide crucial insights into the mechanism of action of this HAE treatment.
(Structure, 3 October 2024)
A clinical evaluation of patients with known mutations (plasminogen and factor XII) with a focus on prophylactic treatment
Robin Lochbaum, et al
The authors analyzed eighteen patients with HAE with normal C1, treated at a clinic in southern Germany. The authors were keen to understand current treatment strategies for these patients, along with disease impact and laboratory values. The authors conclude that all patients received icatibant for on-demand treatment. Additionally, three more patients were given prophylaxis with lanadelumab. In these patients, symptoms and clinical course showed significant improvements.
(Journal of Dermatological Treatment, December 2024)
Long-term safety and efficacy of garadacimab for preventing hereditary angioedema attacks: Phase 3 open-label extension study
Avener Reshef, et al
The authors conducted a clinical trial to investigate the long-term safety and efficacy of garadacimab for HAE prophylaxis. Each patient received treatment for an average of 13.8 months. During this time, 13% of patients reported a side effect from treatment. There were no deaths, and the majority of side effects were mild to moderate. There were three serious side effects reported, but these were not related to garadacimab. The mean HAE attack rate decreased by 95%, and 60% of patients were attack-free. The authors conclude that garadacimab has a favorable safety profile suitable for long-term use and provides long-term protection against HAE attacks.
(Allergy, 7 October 2024)
A quantitative systems pharmacology model of plasma kallikrein-kinin system dysregulation in hereditary angioedema
Dan Sexton, et al
Using computer modeling, the authors developed a virtual patient population to understand the implications of reduced doses of lanadelumab, such as in patients not taking a daily oral dose. The authors conclude that the number of missed doses per month was connected with reduced drug effectiveness. They believe that their model has value in predicting outcomes.
(Journal of Pharmacokinetics and Pharmacodynamics, December 2024)
Recent and anticipated novel drug approvals (3Q 2024 through 2Q 2025)
Matthew H Rim, et al
The authors review a range of new and forthcoming medications for diseases, including HAE. The authors highlight a potential new oral medication for HAE, as part of supporting pharmacists in their role of horizon scanning for future treatments that will need mnaging in formularies and clinical programs.
(American Journal of Health-System Pharmacy, November 2024)
Hereditary angioedema attacks in patients receiving long-term prophylaxis: A systematic review
Hilary J Longhurst, et al
The authors aim to understand what proportion of HAE patients continue to suffer attacks, despite being on long-term prophylaxis. The authors conclude that patients receiving LTP did experience attacks in all parts of the body, including the larynx. Most of these attacks were treated with on-demand therapy, and as a result, the authors feel that access to on-demand therapy remains essential for all people with HAE.
(Clinical Reviews in Allergy & Immunology, 7 November 2024)
Validation and correlations of the Angioedema Activity Score (AAS), Angioedema Quality of Life (AE-QoL) questionnaire, and Angioedema Control Test (AECT) in Chinese patients with angioedema
Hugo WF Mak, et al
The authors aimed to validate the Chinese version of several patient-reported outcome tools, as there had been no previous research to prove these were relevant for Chinese people with angioedema. After testing amongst 118 Chinese patients with recurrent angioedema, the authors conclude that these are valid and reliable tools for use with Chinese patients.
(Journal of Allergy and Clinical Immunology Global, November 2024)
Virtual reality in specialized dentistry: Employing virtual reality for the alleviation of pain and anxiety in hereditary angioedema patients
Alessio Rosa, et al
The authors aimed to use virtual reality technology to address dental anxiety, which may arise in people with HAE following an episode after dental treatment. In the research, people with HAE were given a dental examination while participating in a VR experience, including wearing a headset. The authors conclude that VR can be an excellent distraction tool in patients with HAE who present at least one acute attack per year.
(Minerva Dental and Oral Science, 29 October 2024)
Rare connective tissue diseases in patients with C1-inhibitor deficiency hereditary angioedema: first evidence on prevalence and distribution from a large Italian cohort study
Paola Triggianese, et al
The authors were looking to see the prevalence of rare connective tissue diseases, such as lupus, in people diagnosed with HAE. Examining the data from 855 people with C1 inhibitor deficiency HAE, 2.1% of these people also had a rare connective tissue disease.
(Frontiers in Immunology, 18 October 2024)
Real-world outcomes in patients with hereditary angioedema prescribed lanadelumab versus other prophylaxis
John Anderson, et al
The authors wanted to understand the clinical characteristics, attack history, and quality of life of people with HAE treated with lanadelumab as long-term prophylaxis. In conclusion, the authors write that their findings show that there is a lower attack frequency, lower symptomatic impact, and better quality of life in patients treated with lanadelumab than another prophylaxis in a real-world setting.
(Allergy and Asthma Proceedings, 1 November 2024)
Etiology, recent advances, and clinical trials data for the treatment of angioedema: A review
Mukul Nishad, et al
The authors review recent improvements in knowledge and treatment of various forms of angioedema, including HAE. They conclude that over time outcomes for patients have been greatly improved by a growing understanding of the disease and increasing treatment options.
(Reviews on Recent Clinical Trials, 11 October 2024)
The utility of shared decision making in the management of hereditary angioedema
Rachel Odin, et al
The authors wanted to better understand the patient’s perspective of shared decision making in HAE. They did this by asking 10 US HAE treating physicians, a series of questions following a shared decision making consultation with a patient. The patient was also asked their views and the opinions paired. The authors conclude that shared decisions making is being implemented to determine the best management options for patients with HAE.
(Allergy and Asthma Proceedings, 1 November 2024)
A mechanistic model of in vitro plasma activation to evaluate therapeutic kallikrein-kinin system inhibitors
Alirez Rezvani-Sharif, et al
The authors present a new computer model of the complex biochemical pathway that can go wrong and lead to HAE. The authors feel that their model represents a valuable way to study the interactions within the kallikrein-kinin system, to better understand the ways in which HAE treatments that target this system work.
(PLOS Computational Biology, 4 November 2024)
Initial experience of long-term prophylaxis with lanadelumab for hereditary angioedema in China: A clinical observation study on six patients
Wo Yao, et al
The authors investigated the clinical efficacy and safety of lanadelumab for long-term prophylaxis (LTP) in Chinese people with HAE. The authors followed six Chinese patients treated with lanadelumab, finding that LTP with the medication led to a reduction of 97.8% in attack rate, with no serious or severe treatment-emergent adverse events. The authors conclude that this is the first report to demonstrate the clinical efficacy of lanadalumab and safety of LTP in HAE patients from the Chinese mainland. They suggest that a reasonable dosage plan of lanadelumab can ensure quick and long-lasting protection against HAE attacks.
(International Archives of Allergy and Immunology, 3 October 2024)
Prolonging the circulatory half-life of C1 esterase inhibitor via albumin fusion
Sangavi Sivananthan, et al
The authors investigated how to extend the time existing C1 inhibitor-based treatments last in the body. Their findings, they conclude, suggest that there is potential for their technique to be investigated further.
(PLOS One, 23 October 2024)
Deciphering the landscape of hereditary angioedema in India: Perspective for Indian dermatologists
Prabal Barman, et al
The authors looked into HAE from the dermatologist’s perspective and provided detail on the availability of first-line treatment options in India. The authors encourage their readership to suspect HAE in any patient presenting with swelling without itchy and raised red welts (urticaria). Complement C4, they suggest, offers a simple and sensitive test for HAE. The authors suggest that first-line treatment options for HAE are now available in India and must be used to avoid mortality and improve quality of life.
(Indian Journal of Dermatology, Venereology and Leprology, November-December 2024)
Quantification of C1 inhibitor activity using a chromogenic automated assay: analytical and clinical performances
Yves Renaudineau, et al
Quantifying functional C1 inhibitor activity (fC1-INH) is an important way to diagnose angioedemas such as HAE. The authors tested the performance of an assay of fC1-INH. They conclude that the automated assay tested showed good performance, and could be used both for research and in determining quantitative C1-INH and C4.
(Clinical Chemistry and Laboratory Medicine, 26 November 2024)
Berotralstat for hereditary angioedema with C1 inhibitor deficiency: a practical guide for clinicians
Adil Adatia and Markus Magerl
The authors provide a practical guide for clinicians seeking to use berotralstat in patients with HAE. They conclude that berotralstat is an effective treatment and provides patients with an oral long-term prophylaxis option. Stomach and digestive side effects occur in a minority of patients and can be minimized through the gradual introduction of the medication. Berotralstat may interact with other medications. The authors suggest that an overlap period could prevent breakthrough attacks when switching an HAE patient from plasma-derived C1 inhibitor treatment to berotralstat.
(Frontiers in Immunology, 8 October 2024)
The international HAE guideline under real-life conditions: From possibilities to limits in daily life – current real-world data of 8 German angioedema centers
Jens Greve, et al
The authors aim to exchange knowledge between centers to better address the daily care of people living with HAE. The authors conclude that the current HAE guideline serves as the foundation for how HAE should be treated at specialized centers. However, for rare conditions such as HAE, exchanging information between treating centers can address issues that arise.
(Allergologie Select, 14 November 2024)