dc.contributor
Institut Català de la Salut
dc.contributor
[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
dc.contributor
Vall d'Hebron Barcelona Hospital Campus
dc.contributor.author
Buttgereit, Thomas
dc.contributor.author
Aulenbacher, Felix
dc.contributor.author
Adatia, Adil
dc.contributor.author
Vera Ayala, Carolina
dc.contributor.author
Al-Nesf Al-Mansouri, Maryam Ali Y
dc.contributor.author
Altrichter, Sabine
dc.contributor.author
Guilarte, Mar
dc.date.accessioned
2025-10-24T08:53:30Z
dc.date.available
2025-10-24T08:53:30Z
dc.date.issued
2025-10-06T09:17:31Z
dc.date.issued
2025-10-06T09:17:31Z
dc.date.issued
2025-07-28
dc.identifier
Buttgereit T, Aulenbacher F, Adatia A, Vera Ayala C, Al-Nesf MA, Altrichter S, et al. Unmet needs in hereditary angioedema: an international survey of physicians. Orphanet J Rare Dis. 2025 Jul 28;20:383.
dc.identifier
http://hdl.handle.net/11351/13782
dc.identifier
10.1186/s13023-025-03739-8
dc.identifier
001539225600007
dc.identifier.uri
https://hdl.handle.net/11351/13782
dc.description.abstract
Hereditary angioedema; Management; Treatment goals
dc.description.abstract
Angioedema hereditario; Manejo; Objetivos del tratamiento
dc.description.abstract
Angioedema hereditari; Tractament; Objectius del tractament
dc.description.abstract
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.
dc.description.abstract
Open Access funding enabled and organized by Projekt DEAL. This study was funded by CSL Behring.
dc.format
application/pdf
dc.relation
Orphanet Journal of Rare Diseases;20(1)
dc.relation
https://doi.org/10.1186/s13023-025-03739-8
dc.rights
Attribution 4.0 International
dc.rights
http://creativecommons.org/licenses/by/4.0/
dc.rights
info:eu-repo/semantics/openAccess
dc.subject
Edema - Tractament
dc.subject
Sistema cardiovascular - Malalties - Tractament
dc.subject
ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Epidemiologic Methods::Data Collection::Surveys and Questionnaires
dc.subject
DISEASES::Cardiovascular Diseases::Vascular Diseases::Angioedema::Angioedemas, Hereditary
dc.subject
Other subheadings::Other subheadings::/therapy
dc.subject
TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::métodos epidemiológicos::recopilación de datos::encuestas y cuestionarios
dc.subject
ENFERMEDADES::enfermedades cardiovasculares::enfermedades vasculares::angioedema::angioedemas hereditarios
dc.subject
Otros calificadores::Otros calificadores::/terapia
dc.title
Unmet needs in hereditary angioedema: an international survey of physicians
dc.type
info:eu-repo/semantics/article
dc.type
info:eu-repo/semantics/publishedVersion
