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

Autor: Olivier Fain, Aurelie Du-Thanh, Delphine Gobert, David Launay, Neil Inhaber, Karima Boudjemia, Magali Aubineau, Alain Sobel, Isabelle Boccon-Gibod, Laurence Weiss, Laurence Bouillet
Rok vydání: 2021
Předmět:
Zdroj: Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology. 18(1)
ISSN: 1710-1484
Popis: 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 deviation [SD]) lanadelumab exposure was 240.4 (53.7) days. Lanadelumab dose was modified in 12 patients (mostly to every 4 weeks). For the analyzed population, compared with attacks/month (mean [SD]) within 6 months before ATU (2.68 [2.54]), fewer attacks occurred between initiation and first visit (0.16 [0.42]; P Conclusions Lanadelumab reduced attack rates, improved quality of life, and was generally well tolerated.
Databáze: OpenAIRE