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 for prophylaxis.
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 16 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.
