{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"omics_type":["Unknown"],"volume":["18(1)"],"submitter":["Fain O"],"pubmed_abstract":["<h4>Background</h4>Hereditary angioedema (HAE) is associated with a heavy burden of illness.<h4>Objective</h4>To evaluate use of lanadelumab in a French Authorization for Temporary Use (ATU) program.<h4>Methods</h4>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).<h4>Results</h4>In total, 77 patients received ≥ 1 lanadelumab dose; 69 had ≥ 1 quarterly follow-up visit (analyzed population). Mean (standard de"],"journal":["Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology"],"pagination":["30"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC8976389"],"repository":["biostudies-literature"],"pubmed_title":["Long-term prophylaxis with lanadelumab for HAE: authorization for temporary use in France."],"pmcid":["PMC8976389"],"pubmed_authors":["Boudjemia K","Launay D","Weiss L","Aubineau M","Fain O","Du-Thanh A","Gobert D","Inhaber N","Boccon-Gibod I","Bouillet L","Sobel A"],"additional_accession":[]},"is_claimable":false,"name":"Long-term prophylaxis with lanadelumab for HAE: authorization for temporary use in France.","description":"<h4>Background</h4>Hereditary angioedema (HAE) is associated with a heavy burden of illness.<h4>Objective</h4>To evaluate use of lanadelumab in a French Authorization for Temporary Use (ATU) program.<h4>Methods</h4>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).<h4>Results</h4>In total, 77 patients received ≥ 1 lanadelumab dose; 69 had ≥ 1 quarterly follow-up visit (analyzed population). Mean (standard de","dates":{"release":"2022-01-01T00:00:00Z","publication":"2022 Apr","modification":"2025-06-01T02:44:09.335Z","creation":"2025-06-01T02:44:09.335Z"},"accession":"S-EPMC8976389","cross_references":{"pubmed":["35365234"],"doi":["10.1186/s13223-022-00664-4"]}}