Introduction: Hereditary angioedema (HAE) due to C1 inhibitor (C1-INH) deficiency is a debilitating and potentially lethal disease. Management includes on-demand treatment of angioedema and their prophylaxis. Plasma derived C1-INH is an established treatment for both on demand and prophylaxis of HAE. Conestat alfa is a recombinant form of human C1-INH (rhC1-INH) produced in transgenic rabbits. It has granted drug’s registration as treatment option for acute HAE attacks in adults and adolescents in Europe, America, and other countries. Long-term prophylaxis with rhC1-INH received recent consideration in clinical trials. Areas covered: This review will critically appraise available information about rhC1-INH (conestat alfa) prophylactic treatment in adult and adolescent patients with congenital C1-INH deficiency. Results from a phase II randomized placebo-controlled trial for prophylaxis of severe HAE evidenced positive treatment outcomes for its application, both twice or once weekly. Expert commentary: Phase II clinical studies suggest that rhC1-INH is a viable option for prophylaxis of HAE. Safety and tolerability data are comparable to other available HAE specific drugs, zeroing the possibility for blood-born viral transmission. Sustainability of modern technologies is granting a practically stable and continuous recombinant production process. With other available options, rhC1-INH facilitates tailoring HAE treatment to patients’ needs.
Recombinant human C1 esterase inhibitor (Conestat alfa) for prophylaxis to prevent attacks in adult and adolescent patients with hereditary angioedema / A. Valerieva, S. Caccia, M. Cicardi. - In: EXPERT REVIEW OF CLINICAL IMMUNOLOGY. - ISSN 1744-666X. - 14:9(2018 Sep 18), pp. 707-718.
|Titolo:||Recombinant human C1 esterase inhibitor (Conestat alfa) for prophylaxis to prevent attacks in adult and adolescent patients with hereditary angioedema|
CACCIA, SONIA (Secondo)
CICARDI, MARCO (Ultimo) (Corresponding)
|Parole Chiave:||C1-inhibitor; C1-inhibitor deficiency; conestat alfa; glycosylation; hereditary angioedema; long-term prophylactic treatment; prophylaxis; recombinant C1 inhibitor; rhC1-INH; serpin; Immunology and Allergy; Immunology|
|Settore Scientifico Disciplinare:||Settore MED/09 - Medicina Interna|
Settore BIO/11 - Biologia Molecolare
Settore MED/50 - Scienze Tecniche Mediche Applicate
|Data di pubblicazione:||18-set-2018|
|Digital Object Identifier (DOI):||http://dx.doi.org/10.1080/1744666X.2018.1503055|
|Appare nelle tipologie:||01 - Articolo su periodico|