Sistema de Salut de Catalunya
Institut Català de la Salut
[Buttgereit T, Aulenbacher F, Vera Ayala C] Dermatology and Allergy, Institute of Allergology, Charité–Universitätsmedizin Berlin, Freie Universität Berlin and HumboldtUniversität Zu Berlin, Berlin, Germany. Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Immunology and Allergology, Berlin, Germany. [Adatia A] Division of Pulmonary Medicine, Department of Medicine, University of Alberta, Edmonton, AB, Canada. [Al Nesf MA] Allergy and Immunology Division, Department of Medicine, Hamad Medical Corporation, Doha, Qatar. [Altrichter S] Department of Dermatology and Venereology, Kepler University Hospital, Linz, Austria. Center for Medical Research, Johannes Kepler Universität Linz, Linz, Austria. [Guilarte M] Servei d’Al·lergologia, Vall d’Hebron Hospital Universitari, Barcelona, Spain. Vall d’Hebron Institut de Recerca (VHIR), Barcelona, Spain
Vall d'Hebron Barcelona Hospital Campus
2025-10-06T09:17:31Z
2025-10-06T09:17:31Z
2025-07-28
Hereditary angioedema; Management; Treatment goals
Angioedema hereditario; Manejo; Objetivos del tratamiento
Angioedema hereditari; Tractament; Objectius del tractament
Background: Hereditary angioedema (HAE) is a rare and potentially life-threatening genetic disorder characterized by unpredictable attacks of angioedema. MENTALIST (UnMEt Needs in herediTAry angioedema-a gLobal physIcian perSpecTive) is the first international survey uncovering unmet needs and identifying barriers to optimal management in HAE following the latest update of the World Allergy Organization (WAO)/European Academy of Allergy and Clinical Immunology (EAACI) HAE guidelines. Methods: This web-based survey comprised 24 questions on HAE management and unmet needs. HAE-expert physicians from the Angioedema Centers of Reference and Excellence network ranked unmet needs according to their own perspectives and their patients' perspectives, using a 10-point Likert scale ranging from 0 (not a challenge/unmet need at all) to 10 (huge challenge/unmet need). Results: Of 64 respondents from 32 countries, most (91%) had > 5 years of experience in managing HAE. Overall, 48% of respondents (n = 31/64) reported that < 50% of their patients had achieved the WAO/EAACI HAE treatment goals of total disease control and "normalization" of life at the time of the survey. Implementation of consensus recommendations was found to be inconsistent across regions. Gaps in non-HAE-expert physician knowledge, treatment costs, and reimbursement for long-term prophylaxis were the highest-priority challenges according to the respondents. Burden of disease remains a challenge among patients, as reported by their physicians. Conclusions: The MENTALIST findings highlight a need for removal of barriers to HAE treatment goals and propose a call to action to improve access to treatments, for greater provision of education for physicians and patients, critical collaboration with patient organizations and industry stakeholders and ultimately to optimize HAE care.
Open Access funding enabled and organized by Projekt DEAL. This study was funded by CSL Behring.
Article
Published version
English
Enquestes; Edema - Tractament; Sistema cardiovascular - Malalties - Tractament; ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Epidemiologic Methods::Data Collection::Surveys and Questionnaires; DISEASES::Cardiovascular Diseases::Vascular Diseases::Angioedema::Angioedemas, Hereditary; Other subheadings::Other subheadings::/therapy; TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::métodos epidemiológicos::recopilación de datos::encuestas y cuestionarios; ENFERMEDADES::enfermedades cardiovasculares::enfermedades vasculares::angioedema::angioedemas hereditarios; Otros calificadores::Otros calificadores::/terapia
BMC
Orphanet Journal of Rare Diseases;20(1)
https://doi.org/10.1186/s13023-025-03739-8
Attribution 4.0 International
http://creativecommons.org/licenses/by/4.0/
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