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Pediatric Drugs

, Volume 20, Issue 2, pp 135–151 | Cite as

Pharmacological Management of Hereditary Angioedema with C1-Inhibitor Deficiency in Pediatric Patients

  • Henriette FarkasEmail author
Review Article

Abstract

Hereditary angioedema with C1-inhibitor deficiency (C1-INH-HAE) is a form of bradykinin-mediated angioedema. It is a rare disorder with an onset during childhood in most instances. Therefore, familiarity with the options for the management of pediatric cases is indispensable. The recurrent angioedematous episodes do not respond to conventional treatments and may evolve into a life-threatening condition. In view of the recommendations adopted by international consensus in 2016, patient management and follow-up should be guided by an individualized strategy. During the last decade, various medicinal products with novel modes of action and different posology have been developed for the treatment of C1-INH-HAE. These drugs either inhibit the release of bradykinin (plasma-derived C1-inhibitors, recombinant C1-inhibitors, kallikrein inhibitors) or prevent the released bradykinin from binding to its receptor (bradykinin B2 receptor antagonists). This review summarizes the properties of the medicinal products currently available for the treatment of C1-INH-HAE, the indications for their use in pediatric patients, and the findings of the clinical trials conducted in this patient population. It is concluded by a brief outline of future therapeutic options.

Notes

Acknowledgements

The author wishes to thank Nóra Veszeli, Márta Debreczeni, and Kinga Viktória Kőhalmi PhD students for compiling the list of references to the literature, and for drafting the figure.

Compliance with Ethical Standards

Funding

No sources of funding were used to conduct this study or to prepare this manuscript.

Conflict of interest

The author has received honoraria, travel grants and payment for lectures from CSL Behring, Shire, Swedish Orphan Biovitrum, BioCryst, and Pharming, and/or served as a consultant for these companies.

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© Springer International Publishing AG, part of Springer Nature 2017

Authors and Affiliations

  1. 1.3rd Department of Internal Medicine, Hungarian Angioedema CenterSemmelweis UniversityBudapestHungary

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