Unmet needs in hereditary angioedema: an international survey of physicians
Thomas Buttgereit, Felix Aulenbacher, Adil Adatia, Carolina Vera Ayala, Maryam 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 Betschel, Herberto José Chong‐Neto, Oscar Calderón Llosa, Timothy Craig, Anthony D. Dorr, Sérgio Duarte Dortas, Daria Fomina, Henriette Farkas, Jie Shen Fok, Anete Sevciovic Grumach, Jens Greve, Mar Guilarte, Margarida Gonçalo, Vesna Grivcheva‐Panovska, Michihiro Hide, Roman Hakl, Ankur Kumar Jindal, Constance H. Katelaris, Shailajah Kamaleswaran, Tamar Kinaciyan, Elena A. Latysheva, José Ignacio Larco Sousa, Ramón Lleonart Bellfill, Hassan Mobayed, Martin Metz, Iman Nasr, Natasa Teovska Mitrevska, Stefania Nicola, Claudio Alberto Salvador Parisi, Grzegorz Porębski, Jonny Peter, Mariana Paes Leme Ferriani, Nelson Augusto Rosário Filho, Bülent Enis Şekerel, Faradiba Sarquis Serpa, Marcin Stobiecki, Susanne Trainotti, Anna Valerieva, Chamard Wongsa, Jane C. Y. Wong, Esra Yücel, Yinglei Li, Chiara Nenci, Marcus Maurer, Markus Magerl, Philip H. Li
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.