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...
| Autores: | , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , |
|---|---|
| Tipo de recurso: | artículo |
| Estado: | Versión publicada |
| Fecha de publicación: | 2025 |
| País: | España |
| Institución: | Universidad de Barcelona |
| Repositorio: | Dipòsit Digital de la UB |
| OAI Identifier: | oai:diposit.ub.edu:2445/222842 |
| Acceso en línea: | https://hdl.handle.net/2445/222842 |
| Access Level: | acceso abierto |
| Palabra clave: | Edema angioneuròtic Anafilaxi Angioneurotic edema Anaphylaxis |
| id |
ES_8ed3494a745d20b6ef448db6735feb21 |
|---|---|
| oai_identifier_str |
oai:diposit.ub.edu:2445/222842 |
| network_acronym_str |
ES |
| network_name_str |
España |
| repository_id_str |
|
| spelling |
Unmet needs in hereditary angioedema: an international survey of physiciansButtgereit, ThomasAulenbacher, FelixAdatia, AdilVera Ayala, CarolinaAli Al-Nesf, MaryamAltrichter, SabineAbuzakouk, MohamedAl-Ahmad, MonaMohammed Ali, RamzyBerardi, AlejandroBoccon-Gibod, IsabelleBouillet, LaurenceBrussino, LuisaBarešić, MarkoBusse, Paula J.Betschel, Stephen D.Chong Neto, HerbertoCalderón Llosa, ÓscarCraig, Timothy J.Dorr, Anthony D.Duarte Dortas, SérgioFomina, DariaFarkas, HenrietteShen Fok, JieGrumach, Anete S.Greve, JensGuilarte, MarGonçalo, MargaridaGrivcheva Panovska, VesnaHide, MichihiroHakl, RomanJindal, AnkurKatelaris, Constance H.Kamaleswaran, ShailajahKinaciyan, TamarLatysheva, ElenaLarco Sousa, José IgnacioLleonart Bellfill, RamonMobayed, HassanMetz, MartinNasr, ImanMitrevska, Natasa T.Nicola, StefaniaSalvador Parisi, Claudio AlbertoPorebski, GrzegorzPeter, JonnyFerriani, Mariana Paes LemeRosario, NelsonŞekerel, Bülent EnisSarquis Serpa, FaradibaStobiecki, MarcinTrainotti, SusanneValerieva, AnnaWongsa, ChamardWong, Jane C. Y.Yucel, EsraLi, YingleiNenci, ChiaraMaurer, MarcusMagerl, MarkusLi, Philip H.Edema angioneuròticAnafilaxiAngioneurotic edemaAnaphylaxisBackgroundHereditary 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.Springer Science and Business Media LLC2025info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionapplication/pdfhttps://hdl.handle.net/2445/222842Articles publicats en revistes (Institut d'lnvestigació Biomèdica de Bellvitge (IDIBELL))reponame:Dipòsit Digital de la UBinstname:Universidad de BarcelonaInglésReproducció del document publicat a: https://doi.org/10.1186/s13023-025-03739-8Orphanet Journal of Rare Diseases, 2025, vol. 20, num. 1https://doi.org/10.1186/s13023-025-03739-8cc by (c) Buttgereit, Thomas et al, 2025http://creativecommons.org/licenses/by/3.0/es/info:eu-repo/semantics/openAccessoai:diposit.ub.edu:2445/2228422026-05-27T06:46:51Z |
| dc.title.none.fl_str_mv |
Unmet needs in hereditary angioedema: an international survey of physicians |
| title |
Unmet needs in hereditary angioedema: an international survey of physicians |
| spellingShingle |
Unmet needs in hereditary angioedema: an international survey of physicians Buttgereit, Thomas Edema angioneuròtic Anafilaxi Angioneurotic edema Anaphylaxis |
| title_short |
Unmet needs in hereditary angioedema: an international survey of physicians |
| title_full |
Unmet needs in hereditary angioedema: an international survey of physicians |
| title_fullStr |
Unmet needs in hereditary angioedema: an international survey of physicians |
| title_full_unstemmed |
Unmet needs in hereditary angioedema: an international survey of physicians |
| title_sort |
Unmet needs in hereditary angioedema: an international survey of physicians |
| dc.creator.none.fl_str_mv |
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. |
| author |
Buttgereit, Thomas |
| author_facet |
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. |
| author_role |
author |
| author2 |
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. |
| author2_role |
author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author |
| dc.subject.none.fl_str_mv |
Edema angioneuròtic Anafilaxi Angioneurotic edema Anaphylaxis |
| topic |
Edema angioneuròtic Anafilaxi Angioneurotic edema Anaphylaxis |
| description |
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. |
| publishDate |
2025 |
| dc.date.none.fl_str_mv |
2025 |
| dc.type.none.fl_str_mv |
info:eu-repo/semantics/article info:eu-repo/semantics/publishedVersion |
| format |
article |
| status_str |
publishedVersion |
| dc.identifier.none.fl_str_mv |
https://hdl.handle.net/2445/222842 |
| url |
https://hdl.handle.net/2445/222842 |
| dc.language.none.fl_str_mv |
Inglés |
| language_invalid_str_mv |
Inglés |
| dc.relation.none.fl_str_mv |
Reproducció del document publicat a: https://doi.org/10.1186/s13023-025-03739-8 Orphanet Journal of Rare Diseases, 2025, vol. 20, num. 1 https://doi.org/10.1186/s13023-025-03739-8 |
| dc.rights.none.fl_str_mv |
cc by (c) Buttgereit, Thomas et al, 2025 http://creativecommons.org/licenses/by/3.0/es/ info:eu-repo/semantics/openAccess |
| rights_invalid_str_mv |
cc by (c) Buttgereit, Thomas et al, 2025 http://creativecommons.org/licenses/by/3.0/es/ |
| eu_rights_str_mv |
openAccess |
| dc.format.none.fl_str_mv |
application/pdf |
| dc.publisher.none.fl_str_mv |
Springer Science and Business Media LLC |
| publisher.none.fl_str_mv |
Springer Science and Business Media LLC |
| dc.source.none.fl_str_mv |
Articles publicats en revistes (Institut d'lnvestigació Biomèdica de Bellvitge (IDIBELL)) reponame:Dipòsit Digital de la UB instname:Universidad de Barcelona |
| instname_str |
Universidad de Barcelona |
| reponame_str |
Dipòsit Digital de la UB |
| collection |
Dipòsit Digital de la UB |
| repository.name.fl_str_mv |
|
| repository.mail.fl_str_mv |
|
| _version_ |
1869413161357017088 |
| score |
15,812429 |