Efficacy of lanadelumab in angioedema due to acquired C1 inhibitor deficiency.

J Allergy Clin Immunol Pract

Division of Internal Medicine, ASST Fatebenefratelli-Sacco, Luigi Sacco Hospital, Milan, Italy.

Published: March 2023

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http://dx.doi.org/10.1016/j.jaip.2022.10.041DOI Listing

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Background: Lanadelumab was well tolerated and effective in preventing hereditary angioedema (HAE) attacks in the phase 3, double-blind, placebo-controlled Hereditary angioEdema Long-term Prophylaxis (HELP) study and subsequent HELP open-label extension (OLE) study (NCT02741596).

Objective: To evaluate outcomes from HELP OLE for adolescent patients aged 12 to 17 years.

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Hereditary angioedema (HAE) is a rare genetic disorder characterized by potentially life-threatening episodes of swelling. Most HAE cases are caused by deficient (type I) or dysfunctional (type II) C1-esterase inhibitor (C1-INH) protein. However, some patients present with a subtype of HAE that is associated with normal plasma levels of functional C1-INH protein and complement component 4 (HAE-nC1INH).

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