Unmet needs in hereditary angioedema: an international survey of physicians

Unmet needs in hereditary angioedema: an international survey of physicians

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 a...

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Main Authors: Thomas Buttgereit, Felix Aulenbacher, Adil Adatia, Carolina Vera Ayala, Maryam Ali Al-Nesf, Sabine Altrichter, Mohamed Abuzakouk, Mona Al-Ahmad, Ramzy Mohammed Ali, Alejandro Berardi, Isabelle Boccon-Gibod, Laurence Bouillet, Luisa Brussino, Marko Barešić, Paula J. Busse, Stephen D. Betschel, Herberto Chong-Neto, Oscar Calderón Llosa, Timothy J. Craig, Anthony D. Dorr, Sérgio Duarte Dortas Junior, Daria Fomina, Henriette Farkas, Jie Shen Fok, Anete S. Grumach, Jens Greve, Mar Guilarte, Margarida Gonçalo, Vesna Grivcheva-Panovska, Michihiro Hide, Roman Hakl, Ankur Jindal, Constance H. Katelaris, Shailajah Kamaleswaran, Tamar Kinaciyan, Elena Latysheva, José Ignacio Larco Sousa, Ramón Lleonart Bellfill, Hassan Mobayed, Martin Metz, Iman Nasr, Natasa T. Mitrevska, Stefania Nicola, Claudio Alberto Salvador Parisi, Grzegorz Porebski, Jonny Peter, Mariana Paes Leme Ferriani, Nelson Rosario Filho, Bülent Enis Şekerel, Faradiba Sarquis Serpa, Marcin Stobiecki, Susanne Trainotti, Anna Valerieva, Chamard Wongsa, Jane C. Y. Wong, Esra Yucel, Yinglei Li, Chiara Nenci, Marcus Maurer, Markus Magerl, Philip H. Li
Format: Article
Language:English
Published: BMC 2025-07-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Hereditary angioedema
Guidelines
Management
Treatment goals
Unmet needs
Online Access:https://doi.org/10.1186/s13023-025-03739-8
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Summary: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.
ISSN:1750-1172

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