The symptom experience of hereditary angioedema (HAE) patients beyond HAE attacks: literature review and clinician interviews
Abstract Background Hereditary angioedema (HAE) is a genetic disorder characterized by re-occurring swelling episodes called “attacks,” usually in the limbs, face, airways, and intestinal tract. New prophylactic therapies have reduced the frequency of these attacks. This study describes results from...
| Main Authors: | , , , , |
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| Format: | Article |
| Language: | English |
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BMC
2022-06-01
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| Series: | Orphanet Journal of Rare Diseases |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s13023-022-02360-3 |
| _version_ | 1811341644043124736 |
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| author | Milenka Jean-Baptiste Robbin Itzler Subhransu Prusty Dylan Supina Mona L. Martin |
| author_facet | Milenka Jean-Baptiste Robbin Itzler Subhransu Prusty Dylan Supina Mona L. Martin |
| author_sort | Milenka Jean-Baptiste |
| collection | DOAJ |
| description | Abstract Background Hereditary angioedema (HAE) is a genetic disorder characterized by re-occurring swelling episodes called “attacks,” usually in the limbs, face, airways, and intestinal tract. New prophylactic therapies have reduced the frequency of these attacks. This study describes results from a literature review and clinician interviews assessing patient HAE symptom experiences and timing, and then evaluates whether existing patient-reported outcome (PRO) tools adequately reflect this experience. Methods A targeted literature review as well as interviews with key opinion leaders (KOLs), were conducted to capture information about the patient experience and their symptoms. An assessment of various PROs was then conducted to determine how well they each covered HAE symptoms and impacts. Results Nineteen HAE symptoms were identified. KOLs reported that patients on prophylactic therapy experienced some symptoms indicating an attack was imminent, but then never experienced an attack. The comparison of the different PROs found that the Hereditary Angioedema Patient-Reported Outcome was the instrument that most thoroughly examined the symptoms of patients with HAE. Conclusions Given the introduction of new prophylactic therapies, further research is needed to determine the effect of being attack-free for longer periods of time on health-related quality of life. |
| first_indexed | 2024-04-13T18:58:18Z |
| format | Article |
| id | doaj.art-6940efa64c254708be498255ba7458ec |
| institution | Directory Open Access Journal |
| issn | 1750-1172 |
| language | English |
| last_indexed | 2024-04-13T18:58:18Z |
| publishDate | 2022-06-01 |
| publisher | BMC |
| record_format | Article |
| series | Orphanet Journal of Rare Diseases |
| spelling | doaj.art-6940efa64c254708be498255ba7458ec2022-12-22T02:34:11ZengBMCOrphanet Journal of Rare Diseases1750-11722022-06-0117111210.1186/s13023-022-02360-3The symptom experience of hereditary angioedema (HAE) patients beyond HAE attacks: literature review and clinician interviewsMilenka Jean-Baptiste0Robbin Itzler1Subhransu Prusty2Dylan Supina3Mona L. Martin4EvideraCSL BehringCSL Behring GmbHJazz Pharmaceuticals (Formerly CSL Behring)EvideraAbstract Background Hereditary angioedema (HAE) is a genetic disorder characterized by re-occurring swelling episodes called “attacks,” usually in the limbs, face, airways, and intestinal tract. New prophylactic therapies have reduced the frequency of these attacks. This study describes results from a literature review and clinician interviews assessing patient HAE symptom experiences and timing, and then evaluates whether existing patient-reported outcome (PRO) tools adequately reflect this experience. Methods A targeted literature review as well as interviews with key opinion leaders (KOLs), were conducted to capture information about the patient experience and their symptoms. An assessment of various PROs was then conducted to determine how well they each covered HAE symptoms and impacts. Results Nineteen HAE symptoms were identified. KOLs reported that patients on prophylactic therapy experienced some symptoms indicating an attack was imminent, but then never experienced an attack. The comparison of the different PROs found that the Hereditary Angioedema Patient-Reported Outcome was the instrument that most thoroughly examined the symptoms of patients with HAE. Conclusions Given the introduction of new prophylactic therapies, further research is needed to determine the effect of being attack-free for longer periods of time on health-related quality of life.https://doi.org/10.1186/s13023-022-02360-3Hereditary angioedemaHAE symptoms and impactsC1-INHHRQoLBetween attack symptoms |
| spellingShingle | Milenka Jean-Baptiste Robbin Itzler Subhransu Prusty Dylan Supina Mona L. Martin The symptom experience of hereditary angioedema (HAE) patients beyond HAE attacks: literature review and clinician interviews Orphanet Journal of Rare Diseases Hereditary angioedema HAE symptoms and impacts C1-INH HRQoL Between attack symptoms |
| title | The symptom experience of hereditary angioedema (HAE) patients beyond HAE attacks: literature review and clinician interviews |
| title_full | The symptom experience of hereditary angioedema (HAE) patients beyond HAE attacks: literature review and clinician interviews |
| title_fullStr | The symptom experience of hereditary angioedema (HAE) patients beyond HAE attacks: literature review and clinician interviews |
| title_full_unstemmed | The symptom experience of hereditary angioedema (HAE) patients beyond HAE attacks: literature review and clinician interviews |
| title_short | The symptom experience of hereditary angioedema (HAE) patients beyond HAE attacks: literature review and clinician interviews |
| title_sort | symptom experience of hereditary angioedema hae patients beyond hae attacks literature review and clinician interviews |
| topic | Hereditary angioedema HAE symptoms and impacts C1-INH HRQoL Between attack symptoms |
| url | https://doi.org/10.1186/s13023-022-02360-3 |
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