Current and emerging biologics for the treatment of hereditary angioedema. (3rd June 2019)

• Record Type: Journal Article
• Authors: Perego, Francesca; Wu, Maddalena A.; Valerieva, Anna; Caccia, Sonia; Suffritti, Chiara; Zanichelli, Andrea; Bergamaschini, Luigi; Cicardi, Marco
• Is Part Of: Expert opinion on biological therapy. Volume 19:Number 6(2019)
• Page Start: 517
• Page End: 526
• Publication Date: 2019-06-03
• Subjects: Hereditary angioedema -- rare diseases -- biologics -- treatment -- clinical trial -- chronic disease; Gene therapy -- Periodicals; Protein drugs -- Periodicals; Peptide drugs -- Periodicals; Immunotherapy -- Periodicals; Drug delivery systems -- Periodicals; 615.5
• DOI: 10.1080/14712598.2019.1595581
• Languages: English
• ISSNs: 1471-2598

Abstract

ABSTRACT: Introduction : Hereditary angioedema due to C1-INH deficiency (C1-INH-HAE) is a rare disease with unpredictable, self-limiting and localized swelling episodes involving the cutaneous and subcutaneous tissues. In the last decade, the spectrum of the possibilities to control the disease has considerably changed with the development of biologic therapies making necessary a careful evaluation of the differences among current and emerging treatments to properly optimize the management of patients. Areas covered : This review serves to summarize the literature regarding the use of biologics for the treatment of C1-INH-HAE. Medications already available on the market and new drugs in different phases of development are addressed. Expert opinion : the advent of biologic therapies dramatically improved the lives of patients with C1-INH-HAE although further improvement is still needed. Whether this is cost/effective will be answered in the next years when we will see if these major advances will benefit the majority of the patients. Trial registration: ClinicalTrials.gov identifier: NCT02586805. Trial registration: ClinicalTrials.gov identifier: NCT02741596. Trial registration: ClinicalTrials.gov identifier: NCT03712228. Trial registration: ClinicalTrials.gov identifier: NCT02870972. Trial registration: ClinicalTrials.gov identifier: NCT03485911.