Understanding
Hereditary, Idiopathic, and Acquired Angioedema
Angioedema is a medical condition characterized by localized swelling of the
deeper layers of the skin and mucous membranes. There are three main types of
angioedema based on its underlying cause – hereditary angioedema (HAE),
idiopathic angioedema, and acquired angioedema.
Hereditary angioedema (HAE) is caused due to a deficiency or dysfunction of
C1-esterase inhibitor (C1-INH), a protein that regulates certain inflammatory
pathways. Symptoms typically appear in childhood or adolescence. Idiopathic
angioedema has no identified triggering factor and accounts for majority of
cases. Acquired angioedema is caused due to medications, medical conditions, or
autoimmune disorders that reduce C1-INH levels similar to HAE.
Treatment Goals and Considerations
The principal goals in Angioedema
Treatment include promptly stopping an ongoing attack, preventing
future attacks, managing long-term control, and improving patient’s quality of
life. Treatment options used will depend on the type and severity of attacks,
previous treatment responses, and patient preferences.
Safety and adherence are also important factors to consider given that
angioedema treatments involve lifelong management. Cost and insurance coverage
posed due to the expensive specialty therapies available further influence
treatment decisions. The complexity of the condition requires coordinated care
from specialists.
Prophylactic Therapies
Regular use of prophylactic medications can effectively reduce attack frequency
in HAE patients. The options include C1-INH concentrates administered via
intravenous or subcutaneous routes. A new oral drug, berotralstat, that
inhibits plasma kallikrein is also approved for prophylaxis.
These targeted therapies are highly effective and convenient treatment options.
However, their high drug acquisition costs and need for refrigeration to
preserve shelf life may pose challenges. Anti-fibrinolytics like tranexamic
acid are less expensive alternatives but are associated with risks like blood
clots. Prophylactic attacks are ideally driven by attack history, severity of
symptoms, impact on quality of life etc.
Acute Attack Therapies
For treating acute attacks, available treatment approaches include C1-INH
replacement therapy, bradykinin receptor antagonists, and analgesics. C1-INH
products have emerged as the treatment of choice owing to their superior
efficacy demonstrated in clinical trials. Icatibant, a bradykinin B2 receptor
antagonist, offers a non-blood derived option.
Attack therapies are promptly administered at onset of symptoms to achieve
significantly reduced time to resolution compared to supportive management
alone. Home therapy using self-administered C1-INH concentrates enables faster
treatment and reduces delays. The option chosen will depend on factors like
availability, cost, route of administration etc.
Emerging Treatment Modalities
Novel therapies targeting different pathogenic mechanisms are in the pipeline
to address unmet needs in angioedema management. These include agents targeting
plasmakallikrein, antibodies inhibiting lipopolysaccharide binding protein, and
BDKRB2 gene silencing techniques.
A subcutaneous C1-INH preparation and an oral plasma kallikrein inhibitor are
under regulatory review. Other avenues being explored are immunomodulation
therapies to induce C1-INH tolerance, hematopoietic stem cell therapy to
replace defective cells, and gene therapy techniques to replace defective
genes. Such targeted investigational options hold promise to deliver
significant improvements in effectiveness, quality of life, and therapy
flexibility.
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About Author:
Alice Mutum is a seasoned senior content editor at Coherent
Market Insights, leveraging extensive expertise gained from her previous role
as a content writer. With seven years in content development, Alice masterfully
employs SEO best practices and cutting-edge digital marketing strategies to
craft high-ranking, impactful content. As an editor, she meticulously ensures
flawless grammar and punctuation, precise data accuracy, and perfect alignment
with audience needs in every research report. Alice’s dedication to excellence
and her strategic approach to content make her an invaluable asset in the world
of market insights.
(LinkedIn: www.linkedin.com/in/alice-mutum-3b247b137
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