Efficacy and safety of recombinant human C1-inhibitor for the treatment of attacks of hereditary angioedema: European open-label extension study
| Autor: | Vincenzo Montinaro, Anurag Relan, Shmuel Kivity, Avner Reshef, S. Visscher, Dumitru Moldovan, Enrico Cillari, Elias Toubi, Jose Fabiani, Massimo Triggiani, Marco Cicardi, Andrea Zanichelli, M. Shlesinger, G. Realdi, Mauro Cancian |
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| Jazyk: | angličtina |
| Rok vydání: | 2012 |
| Předmět: |
Adult
Male Adolescent Visual analogue scale Immunology Vial law.invention Young Adult Randomized controlled trial Risk Factors law medicine Humans Immunology and Allergy Clinical significance Young adult Adverse effect Aged business.industry Angioedemas Hereditary Middle Aged medicine.disease Recombinant Proteins Clinical trial Complement Inactivating Agents Treatment Outcome Anesthesia Hereditary angioedema Female business Complement C1 Inhibitor Protein |
| Popis: | SummaryBackground Hereditary angioedema (HAE) owing to C1 inhibitor deficiency is an autosomal dominant disorder, characterized by recurrent, potentially life-threatening, localized attacks of tissue swelling. Current treatment involves the infusion of C1 inhibitor protein (C1-INH) isolated from human plasma. Objectives This open-label extension to a European, Israeli and Argentinean randomized study (NCT00262301) aimed to investigate the efficacy and safety of recombinant human C1 inhibitor (rhC1-INH) as a first-line treatment following an HAE attack, together with its effect on subsequent attacks. Methods An HAE-specific visual analogue scale (VAS) 0–100 mm was used by patients to assess the severity of attack at four anatomical locations. Patients were treated with one, single-vial, fixed-dose of rhC1-INH (2100 U), followed by up to two further vials at the investigators discretion. The primary end-point was the time from first rhC1-INH injection to first onset of relief of symptoms (≥ 20 mm decrease on VAS). Response to treatment was defined as the onset of relief within 4 h. Results A total of 57 patients were treated for 194 HAE attacks. Overall, sustained relief of symptoms was achieved in 87% of rhC1-INH-treated patients within 4 h of treatment, with 57% of attacks requiring only one vial of rhC1-INH. When categorized by successive attacks experienced by individual patients, the response rate to rhC1-INH treatment was 96%, 83%, 87%, 80% and 80% for attacks 1–5 respectively. Treatment with rhC1-INH was well tolerated, with no discontinuations owing to treatment-emergent adverse events and no adverse events relating to immunogenicity. Conclusions and Clinical Relevance Treatment with rhC1-INH provides fast-onset relief for an HAE attack, with a high rate of therapeutic response maintained throughout subsequent attacks. |
| Databáze: | OpenAIRE |
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