Breakthroughs in hereditary angioedema management: a systematic review of approved drugs and those under research
Hereditary angioedema (HAE) is a rare genetic disorder, characterized by recurrent and unexpected potentially life-threatening mucosal swelling. The impairment underlying HAE could be a defect in C1-inhibitor activity, or in its serum concentration. Patients affected by HAE should be treated with on...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
| Published: |
BioExcel Publishing Ltd
2019-10-01
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| Series: | Drugs in Context |
| Subjects: | |
| Online Access: | https://www.drugsincontext.com/breakthroughs-in-hereditary-angioedema-management:-a-systematic-review-of-approved-drugs-and-those-under-research/ |
| _version_ | 1831753154429976576 |
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| author | Stefania Nicola Giovanni Rolla Luisa Brussino |
| author_facet | Stefania Nicola Giovanni Rolla Luisa Brussino |
| author_sort | Stefania Nicola |
| collection | DOAJ |
| description | Hereditary angioedema (HAE) is a rare genetic disorder, characterized by recurrent and unexpected potentially life-threatening mucosal swelling. The impairment underlying HAE could be a defect in C1-inhibitor activity, or in its serum concentration. Patients affected by HAE should be treated with on-demand or prophylactic drugs. Lifelong C1-inhibitor supplementation is sometimes required. In this review, we review the currently approved drugs for HAE due to C1-inhibitor defect and to describe those under research. In particular, we focused on the mechanisms of action, routes of administration, and efficacy of these therapies. A systematic review of the literature was performed using the PubMed database for original articles and clinical trials of HAE treatments from 2005 to 2019. The approved HAE treatments can minimize the risk of death, but they are not effective in complete healing from the disease. The new gene therapies seem to provide promising opportunities for the treatment of hereditary angioedema. However, there are still many unmet needs, including efficacy, route, and timing of administration. |
| first_indexed | 2024-12-21T23:15:54Z |
| format | Article |
| id | doaj.art-187b523f974a42fd98b89ff4af6f7414 |
| institution | Directory Open Access Journal |
| issn | 1740-4398 1740-4398 |
| language | English |
| last_indexed | 2024-12-21T23:15:54Z |
| publishDate | 2019-10-01 |
| publisher | BioExcel Publishing Ltd |
| record_format | Article |
| series | Drugs in Context |
| spelling | doaj.art-187b523f974a42fd98b89ff4af6f74142022-12-21T18:46:55ZengBioExcel Publishing LtdDrugs in Context1740-43981740-43982019-10-01811110.7573/dic.212605Breakthroughs in hereditary angioedema management: a systematic review of approved drugs and those under researchStefania NicolaGiovanni RollaLuisa BrussinoHereditary angioedema (HAE) is a rare genetic disorder, characterized by recurrent and unexpected potentially life-threatening mucosal swelling. The impairment underlying HAE could be a defect in C1-inhibitor activity, or in its serum concentration. Patients affected by HAE should be treated with on-demand or prophylactic drugs. Lifelong C1-inhibitor supplementation is sometimes required. In this review, we review the currently approved drugs for HAE due to C1-inhibitor defect and to describe those under research. In particular, we focused on the mechanisms of action, routes of administration, and efficacy of these therapies. A systematic review of the literature was performed using the PubMed database for original articles and clinical trials of HAE treatments from 2005 to 2019. The approved HAE treatments can minimize the risk of death, but they are not effective in complete healing from the disease. The new gene therapies seem to provide promising opportunities for the treatment of hereditary angioedema. However, there are still many unmet needs, including efficacy, route, and timing of administration.https://www.drugsincontext.com/breakthroughs-in-hereditary-angioedema-management:-a-systematic-review-of-approved-drugs-and-those-under-research/C1-INHC1-inhibitorgene therapyHAEhereditary angioedemalanadelumabserine proteaseSERPING1 |
| spellingShingle | Stefania Nicola Giovanni Rolla Luisa Brussino Breakthroughs in hereditary angioedema management: a systematic review of approved drugs and those under research Drugs in Context C1-INH C1-inhibitor gene therapy HAE hereditary angioedema lanadelumab serine protease SERPING1 |
| title | Breakthroughs in hereditary angioedema management: a systematic review of approved drugs and those under research |
| title_full | Breakthroughs in hereditary angioedema management: a systematic review of approved drugs and those under research |
| title_fullStr | Breakthroughs in hereditary angioedema management: a systematic review of approved drugs and those under research |
| title_full_unstemmed | Breakthroughs in hereditary angioedema management: a systematic review of approved drugs and those under research |
| title_short | Breakthroughs in hereditary angioedema management: a systematic review of approved drugs and those under research |
| title_sort | breakthroughs in hereditary angioedema management a systematic review of approved drugs and those under research |
| topic | C1-INH C1-inhibitor gene therapy HAE hereditary angioedema lanadelumab serine protease SERPING1 |
| url | https://www.drugsincontext.com/breakthroughs-in-hereditary-angioedema-management:-a-systematic-review-of-approved-drugs-and-those-under-research/ |
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