Informace o publikaci

Unmet needs in hereditary angioedema: an international survey of physicians

Autoři	BUTTGEREIT Thomas AULENBACHER Felix ADATIA Adil AYALA Carolina Vera AL-NESF Maryam Ali ALTRICHTER Sabine ABUZAKOUK Mohamed AL-AHMAD Mona ALI Ramzy Mohammed BERARDI Alejandro BOCCON-GIBOD Isabelle BOUILLET Laurence BRUSSINO Luisa BARESIC Marko BUSSE Paula J BETSCHEL Stephen D CHONG-NETO Herberto LLOSA Oscar Calderon CRAIG Timothy J DORR Anthony D JUNIOR Sergio Duarte Dortas FOMINA Daria FARKAS Henriette FOK Jie Shen GRUMACH Anete S GREVE Jens GUILARTE Mar GONCALO Margarida GRIVCHEVA-PANOVSKA Vesna HIDE Michihiro HAKL Roman JINDAL Ankur KATELARIS Constance H KAMALESWARAN Shailajah KINACIYAN Tamar LATYSHEVA Elena SOUSA Jose Ignacio Larco BELLFILL Ramon Lleonart MOBAYED Hassan METZ Martin NASR Iman MITREVSKA Natasa T NICOLA Stefania PARISI Claudio Alberto Salvador POREBSKI Grzegorz PETER Jonny FERRIANI Mariana Paes Leme FILHO Nelson Rosario SEKEREL Buelent Enis SERPA Faradiba Sarquis 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
Rok publikování	2025
Druh	Článek v odborném periodiku
Časopis / Zdroj	Orphanet Journal of Rare Diseases
Fakulta / Pracoviště MU	Lékařská fakulta
Citace
www	https://ojrd.biomedcentral.com/articles/10.1186/s13023-025-03739-8
Doi	https://doi.org/10.1186/s13023-025-03739-8
Klíčová slova	Hereditary angioedema; Guidelines; Management; Treatment goals; Unmet needs
Popis	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.