Poster session

After short closing remarks on the first day by Dr Ankur Jindal, participants moved on to a structured series of 10 short oral poster presentations. The local steering committee of Dr Mar Guilarte, Dr Stefan Cimbollek, and Dr Teresa Caballero moderated these.

Clinical phenotypes and unmet needs in hereditary angioedema

Dr Ayşegül Pehlivanlar Ustaoğlu from the Hacettepe University Hospital in Türkiye gave a brief presentation based on a poster of data among adult HAE patients in Türkiye. The researchers found that 55% had uncontrolled disease and 31% reported depressive symptoms. Overall, the findings showed that HAE is a burden beyond attack frequency and requires comprehensive management.

Additional benefit of switching to donidalorsen for patients with hereditary angioedema having breakthrough attacks: Findings from the OASISplus study

Dr Marta Goyanes-Malumbres from La Paz University Hospital in Spain presented on the relationship between high rates of HAE attacks before treatment with donidalorsen and the impact this had on patients’ sense of disease control and quality of life once they had been switched to donidalorsen. The researchers found that even patients who initially had low rates of HAE attacks reported clinically meaningful improvements in quality of life, emphasizing that working to optimize treatment is important, even in patients who may appear to be well controlled.

Satisfaction with sebetralstat for HAE attacks in patients switching from parenteral on-demand treatments in KONFIDENT-S

Dr Mauro Cancian from the University of Padua in Italy gave a presentation on data from a larger open-label extension study that examined the use of the new HAE treatment sebetralstat in patients who had previously used a different on-demand medication. They found that patients were satisfied with an oral medication and that the time to the beginning of symptom relief was similar, regardless of the previous on-demand medication.

Anxiety in patients using injectable on-demand treatments for hereditary angioedema attacks: Results from an international patient survey

Next to present was Dr Alexis Bocquet from the Grenoble Alpes University Hospital in France. He gave the audience insights from data on anxiety amongst patients using injectable on-demand treatments for HAE attacks. The data suggested that 70% of HAE patients in the study were anxious during their last parenterally treated attack. The authors concluded that oral therapy could potentially address treatment-related anxiety in HAE attacks.

Onvuzosiran (ADX-324), a semiannual SC investigational siRNA targeting prekallikrein for HAE attack prevention

Professor Marc Riedl, from the University of California, San Diego, United States, provided some initial data on a medicine previously known only as ADX-324. The medicine, which could be taken just once every six months, is being studied to prevent attacks. The data showed that the medicine reduced plasma kallikrein levels by at least 80% with a single injection and maintained that reduction over six months. No serious side effects or safety issues were identified, and the medicine continues to be studied.

Exploring the need for short-term prophylaxis in Italian patients treated with lanadelumab: A nationwide real-world study within the ITACA network

Ms Giada De Angeli from the IRCCS Policlinico San Donato in Italy, together with colleagues, investigated the need for short-term prophylaxis in Italian HAE patients receiving long-term prophylaxis with lanadelumab. The research examined medical procedures, including dental treatment, surgeries, and diagnostic tests. Overall, the authors found no significant difference in post-procedure attack rates between procedures performed on lanadelumab patients who received short-term prophylaxis and those who did not.

End of progression of hereditary angioedema attacks with oral deucrictibant immediate-release capsule: RAPIDe-3 phase 3 trial

Professor Marc Riedl returned to present a second poster, focused on the end of progression of an attack, which he suggested is important to patients as it’s the point at which their symptoms stop worsening. He showed that in patients who took deucrictibant, 50% of attacks achieved end of progression in 17.47 minutes (vs. 228.67 minutes for placebo). In 92.8% of attacks, the end of progression came within 12 hours. Dr Riedl suggested that this indicates a rapid response to treatment.

Living with HAE in Hungary: Patients as partners in research

Ms Arianna Kitzinger from HAE Hungary shared data on how people with HAE in Hungary benefit from a strong partnership between the patient organization and Hungarian doctors. She indicated that there are now 222 HAE patients, and 90% of these receive annual check-ups. She walked the audience through 10 pillars of treatment and collaboration, including patient data, treatment and trials, quality of life assessments, and newborn screening. She concluded that the collaboration between Hungarian patients and doctors had advanced HAE research and improved outcomes for people living with HAE.

CHAPTER-1 open-label extension study: Long-term prophylactic treatment with oral deucrictibant improved health-related quality of life in participants with hereditary angioedema

Dr Andrea Zanichelli from IRCCS Policlinico San Donato in Italy presented research into a potential new treatment for HAE. In this research, the focus was on using deucrictibant to prevent HAE attacks, and examined this potential over 134 weeks. He concluded that there were sustained improvements in health-related quality of life for patients, and 100% of those in the extension reported their HAE was well-controlled.

JAV-BARAS: Cost analysis through time-driven activity-based costing (TDABC) for the assistance of patients with hereditary angioedema

In the final short talk poster presentation of the evening, Dr Solange Oliveira Rodrigues Valle from Clementino Fraga Filho University Hospital in Brazil was invited to present research on the costs of care for patients with HAE. She indicated that their data showed the average annual cost for treating patients with HAE was $60,073.81. Of this total, treatment accounted for more than $59,720.44, with diagnosis costing $76.48 and support costing $95.58. She concluded that the annual average cost is high, with most expenses occurring in the treatment phase.