TY - JOUR
T1 - Consensus on treatment goals in hereditary angioedema
T2 - A global Delphi initiative
AU - Maurer, Marcus
AU - Aygören-Pürsün, Emel
AU - Banerji, Aleena
AU - Bernstein, Jonathan A.
AU - Balle Boysen, Henrik
AU - Busse, Paula J.
AU - Bygum, Anette
AU - Caballero, Teresa
AU - Castaldo, Anthony J.
AU - Christiansen, Sandra C.
AU - Craig, Timothy
AU - Farkas, Henriette
AU - Grumach, Anete S.
AU - Hide, Michihiro
AU - Katelaris, Constance H.
AU - Li, H. Henry
AU - Longhurst, Hilary
AU - Lumry, William R.
AU - Magerl, Markus
AU - Martinez-Saguer, Inmaculada
AU - Riedl, Marc A.
AU - Zhi, Yuxiang
AU - Zuraw, Bruce
N1 - Publisher Copyright:
© 2021 The Authors
PY - 2021/12
Y1 - 2021/12
N2 - Background: Hereditary angioedema (HAE) is a rare, life-threatening genetic disorder characterized by recurrent episodes of subcutaneous or submucosal angioedema. The ultimate goals of treatment for HAE remain ill-defined. Objectives: The aim of this Delphi process was to define the goals of HAE treatment and to examine which factors should be considered when assessing disease control and normalization of the patient's life. Methods: The Delphi panel comprised 23 participants who were selected based on involvement with scientific research on HAE or coauthorship of the most recent update and revision of the World Allergy Organization/European Academy of Allergy and Clinical Immunology guideline on HAE. The process comprised 3 rounds of voting. The final round aimed to aggregate the opinions of the expert panel and to achieve consensus. Results: Two direct consensus questions were posed in round 2, based on the responses received in round 1, and the panel agreed that the goals of treatment are to achieve total control of the disease and to normalize the patient's life. For the third round of voting, 21 statements were considered, with the participants reaching consensus on 18. It is clear from the wide-ranging consensus statements that the burdens of disease and treatment should be considered when assessing disease control and normalization of patients’ lives. Conclusions: The ultimate goal for HAE treatment is to achieve no angioedema attacks. The availability of improved treatments and disease management over the last decade now makes complete control of HAE a realistic possibility for most patients.
AB - Background: Hereditary angioedema (HAE) is a rare, life-threatening genetic disorder characterized by recurrent episodes of subcutaneous or submucosal angioedema. The ultimate goals of treatment for HAE remain ill-defined. Objectives: The aim of this Delphi process was to define the goals of HAE treatment and to examine which factors should be considered when assessing disease control and normalization of the patient's life. Methods: The Delphi panel comprised 23 participants who were selected based on involvement with scientific research on HAE or coauthorship of the most recent update and revision of the World Allergy Organization/European Academy of Allergy and Clinical Immunology guideline on HAE. The process comprised 3 rounds of voting. The final round aimed to aggregate the opinions of the expert panel and to achieve consensus. Results: Two direct consensus questions were posed in round 2, based on the responses received in round 1, and the panel agreed that the goals of treatment are to achieve total control of the disease and to normalize the patient's life. For the third round of voting, 21 statements were considered, with the participants reaching consensus on 18. It is clear from the wide-ranging consensus statements that the burdens of disease and treatment should be considered when assessing disease control and normalization of patients’ lives. Conclusions: The ultimate goal for HAE treatment is to achieve no angioedema attacks. The availability of improved treatments and disease management over the last decade now makes complete control of HAE a realistic possibility for most patients.
KW - C1-INH deficiency
KW - Hereditary angioedema
KW - acute treatment
KW - prophylaxis
KW - quality of life
KW - treatment goals
UR - http://www.scopus.com/inward/record.url?scp=85108529057&partnerID=8YFLogxK
U2 - 10.1016/j.jaci.2021.05.016
DO - 10.1016/j.jaci.2021.05.016
M3 - Article
C2 - 34048855
AN - SCOPUS:85108529057
SN - 0091-6749
VL - 148
SP - 1526
EP - 1532
JO - Journal of Allergy and Clinical Immunology
JF - Journal of Allergy and Clinical Immunology
IS - 6
ER -