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.

Current and emerging biologics for the treatment of hereditary angioedema / F. Perego, M.A. Wu, A. Valerieva, S. Caccia, C. Suffritti, A. Zanichelli, L. Bergamaschini, M. Cicardi. - In: EXPERT OPINION ON BIOLOGICAL THERAPY. - ISSN 1471-2598. - 19:6(2019 Mar 26), pp. 517-526.

Current and emerging biologics for the treatment of hereditary angioedema

Wu, Maddalena A;Caccia, Sonia;Suffritti, Chiara;Bergamaschini, Luigi;Cicardi, Marco
2019-03-26

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.
Hereditary angioedema; biologics; chronic disease; clinical trial; rare diseases; treatment
Settore MED/09 - Medicina Interna
Settore BIO/11 - Biologia Molecolare
Settore MED/50 - Scienze Tecniche Mediche Applicate
Settore MED/46 - Scienze Tecniche di Medicina di Laboratorio
26-mar-2019
EXPERT OPINION ON BIOLOGICAL THERAPY
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Utilizza questo identificativo per citare o creare un link a questo documento: http://hdl.handle.net/2434/639145
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