Breakthroughs in hereditary angioedema management: a systematic review of approved drugs and those under research

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Abstract

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.

Keywords: C1-INH, C1-inhibitor, gene therapy, HAE, hereditary angioedema, lanadelumab, serine protease, SERPING1.

Citation: Nicola S, Rolla G, Brussino L. Breakthroughs in hereditary angioedema management: a systematic review of approved drugs and those under research. Drugs in Context 2019; 8: 212605. DOI: 10.7573/dic.212605

Contributions: All authors contributed equally to the preparation of this review. All named authors meet the International Committee of Medical Journal Editors (ICMJE) criteria for authorship for this article, take responsibility for the integrity of the work as a whole, and have given their approval for this version to be published.

Disclosure and potential conflicts of interest: The authors declare that they have no conflicts of interest. The International Committee of Medical Journal Editors (ICMJE) Potential Conflicts of Interests form for the authors is available for download at https://www.drugsincontext.com/wp-content/uploads/2019/09/dic.212605-COI.pdf

Acknowledgments: None.

Funding declaration: There was no funding associated with the preparation of this article.

Copyright: Copyright © 2019 Nicola S, Rolla G, Brussino L. https://doi.org/10.7573/dic.212605. Published by Drugs in Context under Creative Commons License Deed CC BY NC ND 4.0 which allows anyone to copy, distribute, and transmit the article provided it is properly attributed in the manner specified below. No commercial use without permission.

Correct attribution: Copyright © 2019 Nicola S, Rolla G, Brussino L. Published by Drugs in Context under Creative Commons License Deed CC BY NC ND 4.0.

Article URL: https://www.drugsincontext.com/breakthroughs-in-hereditary-angioedema-management:-a-systematic-review-of-approved-drugs-and-those-under-research/

Correspondence: Luisa Brussino, Dipartimento di Scienze Mediche – SSDDU Allergologia e Immunologia Clinica, Università degli Studi di Torino, AO Ordine Mauriziano Umberto I di Torino, C.so Re Umberto, 109 – 10128 Torino, Italy. luisa.brussino@unito.it

Provenance: invited; externally peer reviewed.

Submitted: 19 June 2019; Peer review comments to author: 23 July 2019; Revised manuscript received: 16 August 2019; Accepted: 27 August 2019; Publication date: 2 October 2019.

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