Patient Perspectives on an Optimal Outcome Measure to Assess Efficacy in the Acute Treatment of Hereditary Angioedema Attacks – Marc A Riedl, University of California – San Diego, United States, et al.
Patients were asked to give their feedback on a variety of scales used to measure the patient perspective in a recent clinical trial for sebetralstat, an investigational on-demand (acute) attack therapy. The scales included a visual analog scale (VAS), the patient global impressions of change (PGI-C), and severity (PGI-S). Patients felt all three captured outcomes that were meaningful to people with HAE. The PGI-C scale tracking patient-reported changes in clinical status was preferred over the others. A PGI-C rating of ‘a little better’ was most commonly used to describe the point at which on-demand medication improves an HAE attack.
An International Physician Real-World Practice Patterns Survey Evaluating the Burden Of Illness in Hereditary Angioedema Type I/II – Henriette Farkas, Semmelweis University, Hungary, et al.
In this international survey of physicians, only a third (36%) indicated that their patients/caregivers routinely recorded HAE attacks in a diary. Where diaries aren’t used, at least one characteristic of the attack is recorded in the medical record. However, all physicians indicated that they had at least one adult patient with inadequately controlled HAE. Without better recording of attack data with diaries, it will be difficult to understand the burden of disease and the effectiveness of existing treatments.
Two Ladies with nC1-INH-HAE Suffering from Massive Abdominal Attacks: A Diagnostic and Therapeutic Challenge – Tamar Kinaciyan, Medical University of Vienna, Austria
HAE with normal C1 is very difficult to diagnose, and no medications are licensed to treat attacks for this type of HAE. The authors report that icatibant was
used successfully to treat abdominal attacks in two women where HAE with normal C1 was suspected (as tests for other reasons for swelling were negative).
Investigating the Occurrence of Allergic Diseases in Hungarian Patients with Hereditary Angioedema Due to C1-Inhibitor-Deficiency – Hanga Réka Horváth, ACARE Semmelweis University, Hungary, et al.
In this survey, half (51.4%) of a group of Hungarian HAE patients reported living with allergies (e.g., to pollen, food, or other medications). This is more than twice the national prevalence of allergies (19.3%) in the general population, indicating a connection between allergies and HAE. However, the authors caution that more research is needed.
The Project of Free Diagnosis of Hereditary Angioedema in Ukraine – Anastasiia Bondarenko, P.L. Shupyk National Healthcare University of Ukraine, Ukraine, et al.:
A new project to increase the diagnosis of HAE in Ukraine led to 17 new patients being diagnosed, increasing the number of confirmed cases by a third (34%). The authors indicate that access to laboratory diagnostics is essential to detect HAE and that higher levels of awareness of HAE in doctors lead to improved diagnosis of patients.
>> View abstract: The project of free diagnosis of hereditary angioedema in Ukraine
Development of Two Novel Oral Formulations of a First-in-Class Bradykinin B2 Receptor Antagonist for On-Demand and Prophylactic Treatment of Hereditary Angioedema – Marcus Maurer, Charité – Universitätsmedizin Berlin, Germany, et al.:
Two different oral formulations of an investigational bradykinin-inhibiting HAE compound have shown promise in early laboratory studies. One formulation is to treat attacks, and the other is to prevent attacks. Both are being studied further in ongoing clinical trials for effectiveness and safety.
HAE with Normal C1 Esterase Inhibitor (HAEnC1INH): Diagnosis, Treatment and Attack Frequency from the 2020 Canadian Patient Survey – Jacquie Badiou, HAE Canada, et al.:
This survey of Canadian patients included some who self-reported having HAE with normal C1 levels. Analyzing the data from these patients, there is an average of 14 years between the start of symptoms and diagnosis. Despite diagnosis and access to treatment for HAE, attacks are frequent in these patients, with 47% reporting a throat attack. The authors call for more awareness and targeted treatment for HAE with normal C1.
Hereditary Angioedema with Normal C1 Inhibitor due to FXII Mutation: Report of the First Case Confirmed with Genetic Diagnostic in Chile – Simone Carrère Boronig, Hospital Clínico Universidad de Chile, Chile, et al.:
The authors report the first Chilean case of HAE with normal C1 due to FXII mutation to be confirmed genetically. The patient was tested for HAE, and when these were negative, a genetic study was performed, and the specific mutation was identified.
Registry of Members of The Association of Patients with Hereditary Angioedema of Perú – Oscar Calderón Llosa, ACARE Clínica Sanna el Golf, San Isidro, Perú, and María Margarita Olivares, Clínica Medellín Poblado, Colombia.:
The first Registry of patients with HAE in Perú has identified up to 80 potential individuals. Most (55) are members of HAE Perú (Asociación de Pacientes con Angioedema Hereditario del Perú). Fifteen HAE patients are not members, and ten further patients with HAE symptoms are awaiting the results of laboratory tests. Prevalence estimates indicate there may be up to 700 people with HAE living in Perú.
Quality of Life of Patients with Hereditary Angioedema in Serbia – Markovic Dusanka, University Clinical Center Nis, Serbia, et al.:
Using the Short Form Health Survey (SF-36), the quality of life of a group of HAE patients in Serbia is shown to be significantly affected by the condition, with physical health and pain being significantly worse than in an age and gender-matched group without HAE.
>> View abstract: Quality Of Life Of Patients With Hereditary Angioedema In Serbia
HAE Patients in Ukraine Under War Conditions – Anastasiia Bondarenko, P.L. Shupyk National Healthcare University of Ukraine, Ukraine, et al.:
The migration of people due to the war in Ukraine has significantly impacted access to treatment for Ukrainian people with HAE. Almost a third (30%) of patients reported problems accessing medicine due to the war, and 23% were unable to receive any medicine. (Thankfully) 73% were able to continue to receive medicine from the Ukraine health authority.
>> View abstract: HAE patients in Ukraine under war conditions
Understanding the HAE Patient Journey in Brazil – Raquel de Oliveira Martins, ABRANGHE, The Brazilian Association of Hereditary Angioedema, Brazil, et al.:
A survey of Brazilian HAE patients and caregivers found that only a third (31%) of respondents indicated that they treated all attacks, despite the vast majority (78%) saying that the disease highly impacts quality of life.
>> View abstract: Understanding the HAE Patient Journey in Brazil
The State of Management of HAE in Central America and Caribbean Countries: Results from a Patient Advocacy Lead Survey – Deborah Corcoran, HAE International, et al.:
HAE patient leads, who ultimately represent more than 1,900 people with HAE, report that the disease is under-recognized, under-diagnosed, and under-treated across Central America and the Caribbean. An important step toward improving the lives of people with HAE in Central America and Caribbean countries should involve; each country increasing awareness-raising activities that include engaging with health ministries; and developing data that characterizes the burden of illness and the value of modern HAE therapies.
