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oai:recercat.cat:11351/137822025-10-24T08:53:30Zcom_2072_378070com_2072_378040com_2072_378071col_2072_378092col_2072_378097

2025-10-24T08:53:30Z urn:hdl:11351/13782 Unmet needs in hereditary angioedema: an international survey of physicians Buttgereit, Thomas Aulenbacher, Felix Adatia, Adil Vera Ayala, Carolina Al-Nesf Al-Mansouri, Maryam Ali Y Altrichter, Sabine Guilarte, Mar 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 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. 2025-10-24T08:53:30Z 2025-10-24T08:53:30Z 2025-10-06T09:17:31Z 2025-10-06T09:17:31Z 2025-07-28 info:eu-repo/semantics/article info:eu-repo/semantics/publishedVersion http://hdl.handle.net/11351/13782 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/ info:eu-repo/semantics/openAccess BMC Scientia