Unmet needs in hereditary angioedema: an international survey of physicians

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00159816%3A_____%2F25%3A00082447" target="_blank" >RIV/00159816:_____/25:00082447 - isvavai.cz</a>

Výsledek na webu

<a href="https://link.springer.com/article/10.1186/s13023-025-03739-8" target="_blank" >https://link.springer.com/article/10.1186/s13023-025-03739-8</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1186/s13023-025-03739-8" target="_blank" >10.1186/s13023-025-03739-8</a>

Jazyk výsledku

angličtina

Název v původním jazyce

Unmet needs in hereditary angioedema: an international survey of physicians

Popis výsledku v původním jazyce

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 &gt; 5 years of experience in managing HAE. Overall, 48% of respondents (n = 31/64) reported that &lt; 50% of their patients had achieved the WAO/EAACI HAE treatment goals of total disease control and &quot;normalization&quot; 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.

Název v anglickém jazyce

Unmet needs in hereditary angioedema: an international survey of physicians

Popis výsledku anglicky

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 &gt; 5 years of experience in managing HAE. Overall, 48% of respondents (n = 31/64) reported that &lt; 50% of their patients had achieved the WAO/EAACI HAE treatment goals of total disease control and &quot;normalization&quot; 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.

Druh

J_imp - Článek v periodiku v databázi Web of Science

CEP obor

—

OECD FORD obor

30102 - Immunology

Projekt

—

Návaznosti

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Rok uplatnění

2025

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Název periodika

Orphanet Journal of Rare Diseases

ISSN

—

e-ISSN

1750-1172

Svazek periodika

20

Číslo periodika v rámci svazku

1

Stát vydavatele periodika

GB - Spojené království Velké Británie a Severního Irska

Počet stran výsledku

14

Strana od-do

383

Kód UT WoS článku

001539225600007

EID výsledku v databázi Scopus

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