Treatment with C1-esterase inhibitor concentrate in type I or II hereditary angioedema: A systematic literature review

Abstract

Hereditary angioedema (HAE) due to C1 esterase inhibitor (HAE-C1-INH) deficiency is a rare genetic disorder presenting with recurrent episodes of skin swellings, abdominal pain attacks, and potentially fatal laryngeal edema. This study was designed to review the efficacy and safety of pasteurized, human, plasma-derived C1-INH concentrate for the treatment of patients with HAE-C1-INH. A systematic search of electronic databases up to December 2011 was performed without language or date restrictions. Two reviewers completed the study selection using predefined inclusion criteria, tabulated, and analyzed the data. The data were inappropriate for meta-analysis; thus, a qualitative synthesis was performed. We identified 89 studies (≍2000 patients) that investigated C1-INH. Replacement therapy with C1-INH significantly shortened time to onset of symptom relief in HAE attacks compared with placebo in a randomized controlled trial, and similar improvements were consistently reported in observational and descriptive studies, accompanied by improvements in patients' quality of life. C1-INH has been shown to be effective for patients receiving home therapy and short- and long-term prophylaxis. Treatment with C1-INH was generally well tolerated. Administration of C1-INH was not associated with transmission of viruses or development of autoantibodies irrespective of treatment duration. This research provides additional confirmation of the efficacy of C1-INH in the treatment and prevention of HAE attacks. C1-INH is generally safe and well tolerated and has an excellent safety record for over 25 years of clinical use.

Safety of C1-Esterase Inhibitor in Acute and Prophylactic Therapy of Hereditary Angioedema: Findings from the Ongoing International Berinert Patient Registry

“…The efficacy and safety of C1-INH for treating acute HAE attacks have been well documented in adults, children, and pregnant women. [20][21][22][23][24] A small number of thromboembolic event (TEE) cases have been reported in patients who used C1-INH, generally off label and at supratherapeutic doses, although causality with C1-INH administration is unclear. [25][26][27] As a requirement for US marketing authorization, and to evaluate the continued safety of the product, a registry was established to gather data on treatment with Berinert.…”

Diagnosis and Treatment of Bradykinin-Mediated Angioedema: Outcomes from an Angioedema Expert Consensus Meeting

“…Several treatments exist for HAE types I/II, including C1-INH, the β2 bradykinin receptor antagonist icatibant, and the kallikrein inhibitor ecallantide, which have been successful in the treatment of HAE types I/II for many years [1]. However, for other forms of bradykinin-mediated angioedema, the diagnostic and treatment needs are less well understood.…”

Fulminant systemic capillary leak syndrome due to C1 inhibitor deficiency complicating acute dermatomyositis: a case report

“…Acute treatment of hereditary and acquired angioedema consists of intravenous administration of C1 inhibitor concentrate. Alternatively and in emergency cases, fresh frozen plasma, which contains C1 inhibitor as well, can be transfused [9,13]. In view of the atypical presentation of angioedema in our patient, C1 inhibitor concentrates were not empirically administered although this may have, in retrospect, reversed the capillary leak and that might have saved the patient’s life.…”