Unmet needs in hereditary angioedema: an international survey of physicians
Issued Date
2025-12-01
Resource Type
eISSN
17501172
Scopus ID
2-s2.0-105012460590
Pubmed ID
40722187
Journal Title
Orphanet Journal of Rare Diseases
Volume
20
Issue
1
Rights Holder(s)
SCOPUS
Bibliographic Citation
Orphanet Journal of Rare Diseases Vol.20 No.1 (2025)
Suggested Citation
Buttgereit T., Aulenbacher F., Adatia A., Ayala C.V., Al-Nesf M.A., Altrichter S., Abuzakouk M., Al-Ahmad M., Ali R.M., Berardi A., Boccon-Gibod I., Bouillet L., Brussino L., Barešić M., Busse P.J., Betschel S.D., Chong-Neto H., Llosa O.C., Craig T.J., Dorr A.D., Junior S.D.D., Fomina D., Farkas H., Fok J.S., Grumach A.S., Greve J., Guilarte M., Gonçalo M., Grivcheva-Panovska V., Hide M., Hakl R., Jindal A., Katelaris C.H., Kamaleswaran S., Kinaciyan T., Latysheva E., Sousa J.I.L., Bellfill R.L., Mobayed H., Metz M., Nasr I., Mitrevska N.T., Nicola S., Parisi C.A.S., Porebski G., Peter J., Ferriani M.P.L., Filho N.R., Şekerel B.E., Serpa F.S., Stobiecki M., Trainotti S., Valerieva A., Wongsa C., Wong J.C.Y., Yucel E., Li Y., Nenci C., Maurer M., Magerl M., Li P.H. Unmet needs in hereditary angioedema: an international survey of physicians. Orphanet Journal of Rare Diseases Vol.20 No.1 (2025). doi:10.1186/s13023-025-03739-8 Retrieved from: https://repository.li.mahidol.ac.th/handle/123456789/111622
Title
Unmet needs in hereditary angioedema: an international survey of physicians
Author(s)
Buttgereit T.
Aulenbacher F.
Adatia A.
Ayala C.V.
Al-Nesf M.A.
Altrichter S.
Abuzakouk M.
Al-Ahmad M.
Ali R.M.
Berardi A.
Boccon-Gibod I.
Bouillet L.
Brussino L.
Barešić M.
Busse P.J.
Betschel S.D.
Chong-Neto H.
Llosa O.C.
Craig T.J.
Dorr A.D.
Junior S.D.D.
Fomina D.
Farkas H.
Fok J.S.
Grumach A.S.
Greve J.
Guilarte M.
Gonçalo M.
Grivcheva-Panovska V.
Hide M.
Hakl R.
Jindal A.
Katelaris C.H.
Kamaleswaran S.
Kinaciyan T.
Latysheva E.
Sousa J.I.L.
Bellfill R.L.
Mobayed H.
Metz M.
Nasr I.
Mitrevska N.T.
Nicola S.
Parisi C.A.S.
Porebski G.
Peter J.
Ferriani M.P.L.
Filho N.R.
Şekerel B.E.
Serpa F.S.
Stobiecki M.
Trainotti S.
Valerieva A.
Wongsa C.
Wong J.C.Y.
Yucel E.
Li Y.
Nenci C.
Maurer M.
Magerl M.
Li P.H.
Aulenbacher F.
Adatia A.
Ayala C.V.
Al-Nesf M.A.
Altrichter S.
Abuzakouk M.
Al-Ahmad M.
Ali R.M.
Berardi A.
Boccon-Gibod I.
Bouillet L.
Brussino L.
Barešić M.
Busse P.J.
Betschel S.D.
Chong-Neto H.
Llosa O.C.
Craig T.J.
Dorr A.D.
Junior S.D.D.
Fomina D.
Farkas H.
Fok J.S.
Grumach A.S.
Greve J.
Guilarte M.
Gonçalo M.
Grivcheva-Panovska V.
Hide M.
Hakl R.
Jindal A.
Katelaris C.H.
Kamaleswaran S.
Kinaciyan T.
Latysheva E.
Sousa J.I.L.
Bellfill R.L.
Mobayed H.
Metz M.
Nasr I.
Mitrevska N.T.
Nicola S.
Parisi C.A.S.
Porebski G.
Peter J.
Ferriani M.P.L.
Filho N.R.
Şekerel B.E.
Serpa F.S.
Stobiecki M.
Trainotti S.
Valerieva A.
Wongsa C.
Wong J.C.Y.
Yucel E.
Li Y.
Nenci C.
Maurer M.
Magerl M.
Li P.H.
Author's Affiliation
Universidade de São Paulo
Monash University
The University of Hong Kong
Charité – Universitätsmedizin Berlin
University of Toronto Faculty of Medicine
Cleveland Clinic Foundation
Universidade Federal do Rio de Janeiro
Université Grenoble Alpes
University of Cape Town
Hacettepe Üniversitesi
Masaryk University
Universidade Federal do Parana
Western Sydney University
Postgraduate Institute of Medical Education and Research, Chandigarh
Sechenov First Moscow State Medical University
Semmelweis Egyetem
Johannes Kepler University Linz
Odense Universitetshospital
Jagiellonian University Medical College
Kuwait University
Hospital Universitari de Bellvitge
İstanbul Tıp Fakültesi
Pirogov Russian National Research Medical University (RNRMU)
Universitätsklinikum Ulm
Queen Mary Hospital Hong Kong
Siriraj Hospital
University of Alberta, Faculty of Medicine and Dentistry
Università degli Studi di Torino, Scuola di Medicina
KBC Zagreb
Ss. Cyril and Methodius University in Skopje
İstanbul University-Cerrahpaşa Cerrahpaşa Faculty of Medicine
Barts Health NHS Trust
Monash Medical Centre
Centro Hospitalar e Universitário de Coimbra
Hamad Medical Corporation
TUM Fakultät für Medizin
Hospital Italiano de Buenos Aires
Vall d'Hebron Institut de Recerca
Fakultní Nemocnice u Sv. Anny v Brně
Hiroshima University Hospital
Kepler Universitätsklinikum
Centro Universitário FMABC
University Hospital Alexandrovska
Medizinische Universität Wien Universitätsklinik für Dermatologie
Cleveland Clinic Abu Dhabi
Hiroshima City Hiroshima Citizens Hospital
Royal Hospital
Box Hill Hospital
Fraunhofer Institute for Translational Medicine and Pharmacology ITMP
Astana Medical University
University of Cape Town Lung Institute
Escola Superior de Ciencias da Santa Casa de Misericordia de Vitoria
CSL Behring
Clínica San Felipe
Re-Medika
Division of Clinical Immunology
SANNA ElGolf Clinic
Scientific and Clinical Consulting Center of Allergology and Immunology
Vinmec International Hospital
CSL Behring
GA²LEN ACARE Angioedema Centers of Reference and Excellence
Monash University
The University of Hong Kong
Charité – Universitätsmedizin Berlin
University of Toronto Faculty of Medicine
Cleveland Clinic Foundation
Universidade Federal do Rio de Janeiro
Université Grenoble Alpes
University of Cape Town
Hacettepe Üniversitesi
Masaryk University
Universidade Federal do Parana
Western Sydney University
Postgraduate Institute of Medical Education and Research, Chandigarh
Sechenov First Moscow State Medical University
Semmelweis Egyetem
Johannes Kepler University Linz
Odense Universitetshospital
Jagiellonian University Medical College
Kuwait University
Hospital Universitari de Bellvitge
İstanbul Tıp Fakültesi
Pirogov Russian National Research Medical University (RNRMU)
Universitätsklinikum Ulm
Queen Mary Hospital Hong Kong
Siriraj Hospital
University of Alberta, Faculty of Medicine and Dentistry
Università degli Studi di Torino, Scuola di Medicina
KBC Zagreb
Ss. Cyril and Methodius University in Skopje
İstanbul University-Cerrahpaşa Cerrahpaşa Faculty of Medicine
Barts Health NHS Trust
Monash Medical Centre
Centro Hospitalar e Universitário de Coimbra
Hamad Medical Corporation
TUM Fakultät für Medizin
Hospital Italiano de Buenos Aires
Vall d'Hebron Institut de Recerca
Fakultní Nemocnice u Sv. Anny v Brně
Hiroshima University Hospital
Kepler Universitätsklinikum
Centro Universitário FMABC
University Hospital Alexandrovska
Medizinische Universität Wien Universitätsklinik für Dermatologie
Cleveland Clinic Abu Dhabi
Hiroshima City Hiroshima Citizens Hospital
Royal Hospital
Box Hill Hospital
Fraunhofer Institute for Translational Medicine and Pharmacology ITMP
Astana Medical University
University of Cape Town Lung Institute
Escola Superior de Ciencias da Santa Casa de Misericordia de Vitoria
CSL Behring
Clínica San Felipe
Re-Medika
Division of Clinical Immunology
SANNA ElGolf Clinic
Scientific and Clinical Consulting Center of Allergology and Immunology
Vinmec International Hospital
CSL Behring
GA²LEN ACARE Angioedema Centers of Reference and Excellence
Corresponding Author(s)
Other Contributor(s)
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.