Long-term prophylaxis with lanadelumab for HAE: authorization for temporary use in France.

ABSTRACT:

Background

Hereditary angioedema (HAE) is associated with a heavy burden of illness.

Objective

To evaluate use of lanadelumab in a French Authorization for Temporary Use (ATU) program.

Methods

ATU requests were made between October 12, 2018, and March 13, 2019; patients were followed through September 23, 2019. At entry, patients received lanadelumab 300 mg every 2 weeks. HAE attack characteristics were evaluated at day (D) 0 and months (M) 3 and 6. Patients completed the Angioedema Quality of Life (AE-QoL) questionnaire at initiation and monthly and the Angioedema Activity Score questionnaire daily in 28 day cycles (AAS28).

Results

In total, 77 patients received ≥ 1 lanadelumab dose; 69 had ≥ 1 quarterly follow-up visit (analyzed population). Mean (standard de

SUBMITTER: Fain O 

PROVIDER: S-EPMC8976389 | biostudies-literature | 2022 Apr

REPOSITORIES: biostudies-literature