Alexander Troelnikov MBBS , Karen Milburn RN , Pravin Hissaria MBBS, MD , Thanh Thao (Adriana) Le MBBS , William Smith MBBS, PhD
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引用次数: 0
Abstract
Background
Hereditary angioedema (HAE) due to deficiency of C1 Inhibitor (C1INH-HAE) is a rare, unpredictable and potentially fatal genetic disorder. There are relatively few systematic population prevalence studies, with reports from various countries of between 1 in 20,000 and 1 in 150,000. and no Australian data. The therapeutic landscape for HAE has changed dramatically in recent years with a focus on highly effective prophylaxis, with the aim of total suppression of angioedema and achievement of a normal life.
Objectives
Epidemiological survey of HAE in South Australia, with description of patient characteristics, quality of life and treatment, with a focus on prophylaxis.
Methods
Case ascertainment was conducted over 18 months from January 2021 to July 2022, using a range of approaches with the aim of identifying all people with C1INH-HAE in South Australia. Questionnaires were administered to consenting patients utilising established HAE-specific and general survey instruments.
Results
We identified 35 people with HAE in South Australia, yielding a population prevalence of 1 in 52,400, in line with average established international prevalence. HAE was identified in 4 patients of Indigenous Australian heritage. Seventeen of 31 adult patients completed an additional multi-questionnaire survey, revealing overall satisfactory disease control. Most common prophylactic therapies were danazol, lanadelumab, and subcutaneous C1 inhibitor. Many patients (mostly male) with milder disease had responded well to low-dose danazol with good tolerance and have continued to use it, whereas patients with higher disease burden are now using newer therapies, and overall satisfaction with current prophylaxis is high.
Conclusions
Prevalence of HAE in South Australia aligns with international reports. Our population survey indicates that current long-term prophylaxis therapies including danazol, lanadelumab and C1-inhibitor, applied to appropriate patients taking into account disease activity and drug risks and tolerance, are effective for HAE attack prevention and produce high levels of satisfaction.
期刊介绍:
The official pubication of the World Allergy Organization, the World Allergy Organization Journal (WAOjournal) publishes original mechanistic, translational, and clinical research on the topics of allergy, asthma, anaphylaxis, and clincial immunology, as well as reviews, guidelines, and position papers that contribute to the improvement of patient care. WAOjournal publishes research on the growth of allergy prevalence within the scope of single countries, country comparisons, and practical global issues and regulations, or threats to the allergy specialty. The Journal invites the submissions of all authors interested in publishing on current global problems in allergy, asthma, anaphylaxis, and immunology. Of particular interest are the immunological consequences of climate change and the subsequent systematic transformations in food habits and their consequences for the allergy/immunology discipline.