Unmet needs in hereditary angioedema: an international survey of physicians

BackgroundHereditary 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 identif...

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Detalles Bibliográficos
Autores: Buttgereit, Thomas, Aulenbacher, Felix, Adatia, Adil, Vera Ayala, Carolina, Ali Al-Nesf, Maryam, Altrichter, Sabine, Abuzakouk, Mohamed, Al-Ahmad, Mona, Mohammed Ali, Ramzy, Berardi, Alejandro, Boccon-Gibod, Isabelle, Bouillet, Laurence, Brussino, Luisa, Barešić, Marko, Busse, Paula J., Betschel, Stephen D., Chong Neto, Herberto, Calderón Llosa, Óscar, Craig, Timothy J., Dorr, Anthony D., Duarte Dortas, Sérgio, Fomina, Daria, Farkas, Henriette, Shen Fok, Jie, Grumach, Anete S., Greve, Jens, Guilarte, Mar, Gonçalo, Margarida, Grivcheva Panovska, Vesna, Hide, Michihiro, Hakl, Roman, Jindal, Ankur, Katelaris, Constance H., Kamaleswaran, Shailajah, Kinaciyan, Tamar, Latysheva, Elena, Larco Sousa, José Ignacio, Lleonart Bellfill, Ramon, Mobayed, Hassan, Metz, Martin, Nasr, Iman, Mitrevska, Natasa T., Nicola, Stefania, Salvador Parisi, Claudio Alberto, Porebski, Grzegorz, Peter, Jonny, Ferriani, Mariana Paes Leme, Rosario, Nelson, Şekerel, Bülent Enis, Sarquis Serpa, Faradiba, Stobiecki, Marcin, Trainotti, Susanne, Valerieva, Anna, Wongsa, Chamard, Wong, Jane C. Y., Yucel, Esra, Li, Yinglei, Nenci, Chiara, Maurer, Marcus, Magerl, Markus, Li, Philip H.
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2025
País:España
Institución:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
Repositorio:Recercat. Dipósit de la Recerca de Catalunya
OAI Identifier:oai:recercat.cat:2445/222842
Acceso en línea:https://hdl.handle.net/2445/222842
Access Level:acceso abierto
Palabra clave:Edema angioneuròtic
Anafilaxi
Angioneurotic edema
Anaphylaxis
Descripción
Sumario:BackgroundHereditary 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.MethodsThis 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).ResultsOf 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.ConclusionsThe 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.

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