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Yuxiang Zhi
Corresponding Author
ORCiD: https://orcid.org/0000-0001-7539-6650
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This work is licensed under a CC BY 4.0 License. Read Full License
Background: Hereditary angioedema (HAE) is a rare disease with potential life-threatening risks. To study the natural course of HAE under therapy-free conditions throughout patient life is essential for practitioners and patients to avoid possible risk factors and guide treatment.
Objectives: Describe the natural course of HAE and explore possible risk factors, providing new clues for guiding clinical prevention and treatment.
Methods: A web-based survey was conducted in 103 Chinese patients with type 1 HAE. Disease progression at different age stages was provided by each participant. The data for exploring the natural course of HAE composed of two parts: one came from the participants who had never adopted any prophylactic drug for HAE; the other was from the patients with a history of medication, but only the periods before they got confirmed diagnosis and received medications were analyzed. The demographic characteristics, lifestyles, disease severity, and family history were also collected.
Results: Among 103 patients, 14 (13.6%) had their first HAE attack before ten years old and 51 (49.5%) between 10 and 19. The disease worsened in 83.3% of the patients in their twenties. The proportion of patients with symptoms alleviated increased after the age of 30 years old, but the disease maintained relatively severe in most cases before 50. The participants also reported 233 members shared similar symptoms of angioedema in their family and 30 had died of laryngeal edema with the median death age of 46 years old. The disease severity was not observed to be affected significantly by gender, BMI, alcohol or smoking.
Conclusions: We summarized HAE progression patterns under therapy-free conditions, showing the natural course of HAE development along with aging. Long-term prophylaxis and symptomatic treatment are recommended for all HAE patients, especially young and middle-aged and might be adjusted depending on the disease progression.
Keywords
hereditary angioedema (HAE), natural course, risk factors
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CURRENT STATUS: Published
View the published article at Orphanet Journal of Rare Diseases.
Version 3
Posted 28 Aug, 2020
No community comments so far
Editorial decision: Accept
On 04 Sep, 2020
Editor assigned
On 26 Aug, 2020
Submission checks complete
On 25 Aug, 2020
Editor invited
On 25 Aug, 2020
No comments provided
Reviewer #1 agreed
On 18 Aug, 2020
Review #1 received
Received 18 Aug, 2020
Reviewers invited
Invitations sent on 17 Aug, 2020
Editor assigned
On 16 Aug, 2020
Submission checks complete
On 15 Aug, 2020
Editor invited
On 15 Aug, 2020
No comments provided
Editorial decision: Major revision
On 02 Aug, 2020
Reviewer #2 agreed
On 24 Jul, 2020
Review #2 received
Received 24 Jul, 2020
Review #1 received
Received 26 Jun, 2020
Reviewer #1 agreed
On 11 Jun, 2020
Reviewers invited
Invitations sent on 08 Jun, 2020
First submitted
On 13 May, 2020
Editor assigned
On 13 May, 2020
Submission checks complete
On 12 May, 2020
Editor invited
On 12 May, 2020
Metrics
Comments: 0
PDF Downloads: ...
HTML Views: ...
Subject Areas
Internal Medicine
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A new preprint design is coming!
We have improved readability, clarity, accessibility, and opportunities for engagement.
See the published version of this preprint at Orphanet Journal of Rare Diseases.
This is a preprint, a preliminary version of a manuscript that has not completed peer review at a journal. Research Square does not conduct peer review prior to posting preprints. The posting of a preprint on this server should not be interpreted as an endorsement of its validity or suitability for dissemination as established information or for guiding clinical practice.
Learn more about In Review
Research
Yuxiang Zhi
Corresponding Author
ORCiD: https://orcid.org/0000-0001-7539-6650
Badges
Prescreen
This manuscript passed our Prescreen assessment
Complete author information
Appropriate declaration statements
Potential risks to human health
Prescreen
Peer Review Timeline
CURRENT STATUS: Published
View the published article at Orphanet Journal of Rare Diseases.
Version 3
Posted 28 Aug, 2020
No community comments so far
Editorial decision: Accept
On 04 Sep, 2020
Editor assigned
On 26 Aug, 2020
Submission checks complete
On 25 Aug, 2020
Editor invited
On 25 Aug, 2020
No comments provided
Reviewer #1 agreed
On 18 Aug, 2020
Review #1 received
Received 18 Aug, 2020
Reviewers invited
Invitations sent on 17 Aug, 2020
Editor assigned
On 16 Aug, 2020
Submission checks complete
On 15 Aug, 2020
Editor invited
On 15 Aug, 2020
No comments provided
Editorial decision: Major revision
On 02 Aug, 2020
Reviewer #2 agreed
On 24 Jul, 2020
Review #2 received
Received 24 Jul, 2020
Review #1 received
Received 26 Jun, 2020
Reviewer #1 agreed
On 11 Jun, 2020
Reviewers invited
Invitations sent on 08 Jun, 2020
First submitted
On 13 May, 2020
Editor assigned
On 13 May, 2020
Submission checks complete
On 12 May, 2020
Editor invited
On 12 May, 2020
Metrics
Comments: 0
PDF Downloads: ...
HTML Views: ...
Subject Areas
Internal Medicine
License:
This work is licensed under a CC BY 4.0 License. Read Full License
View the published article at Orphanet Journal of Rare Diseases.
Background: Hereditary angioedema (HAE) is a rare disease with potential life-threatening risks. To study the natural course of HAE under therapy-free conditions throughout patient life is essential for practitioners and patients to avoid possible risk factors and guide treatment.
Objectives: Describe the natural course of HAE and explore possible risk factors, providing new clues for guiding clinical prevention and treatment.
Methods: A web-based survey was conducted in 103 Chinese patients with type 1 HAE. Disease progression at different age stages was provided by each participant. The data for exploring the natural course of HAE composed of two parts: one came from the participants who had never adopted any prophylactic drug for HAE; the other was from the patients with a history of medication, but only the periods before they got confirmed diagnosis and received medications were analyzed. The demographic characteristics, lifestyles, disease severity, and family history were also collected.
Results: Among 103 patients, 14 (13.6%) had their first HAE attack before ten years old and 51 (49.5%) between 10 and 19. The disease worsened in 83.3% of the patients in their twenties. The proportion of patients with symptoms alleviated increased after the age of 30 years old, but the disease maintained relatively severe in most cases before 50. The participants also reported 233 members shared similar symptoms of angioedema in their family and 30 had died of laryngeal edema with the median death age of 46 years old. The disease severity was not observed to be affected significantly by gender, BMI, alcohol or smoking.
Conclusions: We summarized HAE progression patterns under therapy-free conditions, showing the natural course of HAE development along with aging. Long-term prophylaxis and symptomatic treatment are recommended for all HAE patients, especially young and middle-aged and might be adjusted depending on the disease progression.
Figure 1
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