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Efficacy of lanadelumab, a monoclonal antibody to plasma kallikrein, in patients with Hereditary Angioedema Type I and II, outside of controlled clinical trials
Introduction Patients with hereditary angioedema (HAE) suffer from recurrent swelling of the skin and mucous membranes of different frequencies, which may also be life threatening if the airway is involved. In severely affected HAE patients, prophylactic therapy is used in addition to a well-effective on-demand therapy. The new monoclonal antibody to plasma kallikrein, lanadelumab, has shown good efficacy in the marketing authorisation studies and can be administered subcutaneously at a low frequency compared to existing therapies.
Material and Methods For the first time outside a controlled trial, we conducted a prospective evaluation of disease-related quality of life (AE-QoL) and angioedema attack frequency and severity in patients with HAE type I / II who have been treated for prophylactic therapy with lanadelumab.
Results A total of 12 patients were placed on prophylactic therapy with lanadelumab. For the majority of these patients, there was no breakthrough attack during the 6-month follow-up period. Disease-related quality of life also improved significantly and relevant side effects were not observed.
Conclusion Prophylactic therapy with lanadelumab is a highly effective therapy with no relevant side effects in patients with HAE Type I / II, even outside controlled clinical trials.
Article published online:
10 June 2020
© 2020. The Author(s). This is an open access article published by Thieme under the terms of the Creative Commons Attribution-NonDerivative-NonCommercial-License, permitting copying and reproduction so long as the original work is given appropriate credit. Contents may not be used for commercial purposes, or adapted, remixed, transformed or built upon. (https://creativecommons.org/licenses/by-nc-nd/4.0/).
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