Basic and clinical immunology
Hereditary angioedema: A decade of human C1-inhibitor concentrate therapy

https://doi.org/10.1016/j.jaci.2007.06.026Get rights and content

Background

C1-inhibitor (C1-INH) is a serine protease inhibitor regulating the complement, kinin-kallikrein, coagulation, and fibrinolytic systems. Hereditary angioedema (HAE) is caused by an inherited deficiency of C1-INH characterized by sudden, recurrent edematous swellings of the subcutaneous or submucosal tissues. The optional therapy for the acute management of HAE is administration of human C1-INH (hC1-INH) concentrate. However, hC1-INH is not available in many countries, in which case fresh frozen plasma is an alternative.

Objective

To summarize our experience with hC1-INH concentrate in patients with HAE.

Methods

Clinical and laboratory information on the effectiveness and safety of hC1-INH administered to relieve 468 acute edematous attacks in 61 patients with HAE was analyzed.

Results

Severe abdominal or subcutaneous attacks and laryngeal edema were consistently relieved by the administration of 500 U hC1-INH concentrate. Symptoms improved within 15 to 60 minutes of administration. Progression of the attacks was never observed, and there were no recurrent attacks within 72 hours. hC1-INH concentrate requirements did not change after repeated use. hC1-INH concentrate proved effective in the management of 94 attacks in 22 children and 6 attacks in 4 pregnant women. Adverse reactions, viral infections, and antibody formation against the purified protein did not occur.

Conclusion

The administration of hC1-INH concentrate in HAE is highly effective and safe for the treatment of acute attacks and short-term prophylaxis and in pediatric patients and pregnant women.

Clinical implications

Human C1-INH concentrate is effective and safe for the treatment of acute HAE attacks as well as for short-term prophylaxis.

Section snippets

Patients

Relevant data of 112 patients treated at the center18 since 1996 were analyzed (Table I). At the initial visit, patients diagnosed with HAE—established by clinical manifestations and complement screening—were invited by mail to attend control visits, scheduled at 1-year intervals at least. The disease severity class of patients was determined according to the criteria for evaluating disease severity, developed by experts from the Novel Methods for Predicting, Preventing, and Treating Attacks in

Results

Of the 112 individuals managed at the center, 61 (including 22 children) had received hC1-INH concentrate intravenously during their life. Fifty-one patients had never been treated with this agent, and 5 had never had an edematous episode. These 2 subsets (ie, patients treated vs not treated with hC1-INH concentrate) were similar with regard to age, sex, ratio of children/adults, type of HAE, and the time of onset of initial symptoms. In the subset treated with hC1-INH concentrate, HAE was

Discussion

In many countries, C1-INH concentrate is still unavailable; therefore, patients with HAE receive either symptomatic treatments22 or fresh frozen plasma.23, 24, 25 However, the use of fresh frozen plasma is associated with a relatively high risk of alloimmunization,26 anaphylactoid reactions,27 transfer of viral infections,28 and transfusion-related acute lung injury.29 Moreover, human plasma contains a variety of components that are undesirable during use for this purpose. Administration of a

References (40)

Cited by (118)

  • Experience with Intravenous Plasma-Derived C1-Inhibitor in Pregnant Women with Hereditary Angioedema: A Systematic Literature Review

    2020, Journal of Allergy and Clinical Immunology: In Practice
    Citation Excerpt :

    Of these, 69 articles were excluded because they did not have data about C1-INH use during pregnancy (51 articles) or were not written in English (16 articles). Two additional articles were preliminary reports of cases21,22 that were more completely described in later reports,17,23 so the preliminary reports were excluded. Of the 16 articles that were not written in English, 5 had no associated abstract, and 8 had an English abstract that did not include data about the use of C1-INH in pregnancy.

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Disclosure of potential conflict of interest: The authors have declared that they have no conflict of interest.

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