A Core Outcome Set for Efficacy of Acute Treatment of Hereditary Angioedema

被引：3

作者：

Petersen, Remy S. ^{[1
]}

Fijen, Laure M. ^{[1
]}

Apfelbacher, Christian ^{[2
]}

Magerl, Markus ^{[3
,4
,5
,6
]}

Weller, Karsten ^{[3
,4
,5
,6
]}

Aberer, Werner ^{[7
]}

Adatia, Adil ^{[8
]}

Audhya, Paul ^{[9
]}

Bara, Noemi-Anna ^{[10
]}

Betschel, Stephen ^{[11
]}

Boccon-Gibod, Isabelle ^{[11
]}

Bouillet, Laurence ^{[12
,14
]}

Brodszki, Nicholas ^{[13
]}

Busse, Paula J. ^{[15
]}

Buttgereit, Thomas ^{[3
,4
]}

Bygum, Anette ^{[16
]}

Cancian, Mauro ^{[17
]}

Craig, Timothy ^{[18
]}

Csuka, Dorottya ^{[19
]}

Farkas, Henriette ^{[19
]}

Fomina, Daria ^{[20
,21
,22
]}

Gil-Serrano, Johana ^{[23
,24
]}

Gompels, Mark ^{[25
]}

Fogelbach, Guillermo Guidos ^{[26
]}

Guilarte, Mar ^{[23
,24
]}

Hide, Michihiro ^{[27
]}

Kiani-Alikhan, Sorena ^{[28
]}

Kinaciyan, Tamar ^{[29
]}

Lenten, Annet ^{[30
,43
]}

Lleonart, Ramon ^{[31
]}

Longhurst, Hilary ^{[32
,33
]}

Lumry, William R. ^{[34
]}

Malbran, Alejandro ^{[35
]}

Malinauskiene, Laura ^{[33
]}

Campos, Juan J. Matta ^{[36
,37
]}

Mendivil, Joan ^{[38
]}

Nieto-Martinez, Sandra A. ^{[39
]}

Peter, Jonathan G. ^{[40
,41
]}

Porebski, Grzegorz ^{[42
]}

Reshef, Avner ^{[39
]}

Riedl, Marc ^{[44
]}

Valerieva, Anna ^{[45
,46
]}

Waserman, Susan ^{[47
]}

Maurer, Marcus ^{[3
,4
]}

Cohn, Danny M. ^{[3
,4
,5
]}

机构：

[1] Univ Amsterdam, Dept Vasc Med, Amsterdam Cardiovasc Sci, Amsterdam UMC, Amsterdam, Netherlands

[2] Otto von Guericke Univ, Inst Social Med & Hlth Syst Res, Med Fac, Magdeburg, Germany

[3] Charite Univ Medi Berlin, Inst Allergol, Angioedema Ctr Reference & Excellence, Berlin, Germany

[4] Free Univ Berlin, Berlin, Germany

[5] Humboldt Univ, Berlin, Germany

[6] Fraunhofer Inst Translat Med & Pharmacol ITMP, Immunol & Allergol, Berlin, Germany

[7] Med Univ Graz, Dept Dermatol, Graz, Austria

[8] Univ Alberta, Dept Med, Div Pulm Med, Edmonton, AB, Canada

[9] KalVista Pharmaceut, Cambridge, MA USA

[10] Mediquest Clin Res Ctr, Romanian Hereditary Angioedema Expertise Ctr, Sangeorgiu De Mures, Romania

[11] Univ Toronto, St Michaels Hosp, Dept Med, Toronto, ON, Canada

[12] CHU Grenoble Alpes, Natl Reference Ctr Angioedema CREAK, Angioedema Ctr Reference & Excellence, Echirolles, France

[13] Univ Grenoble Alpes, CNRS, UMR 5525, VetAgro Sup,Grenoble INP,CHU Grenoble Alpes,TIMC, Grenoble, France

[14] Skane Univ Hosp, Childrens Hosp, Dept Pediat Immunol, Lund, Sweden

[15] Icahn Sch Med Mt Sinai, Div Allergy & Clin Immunol, New York, NY USA

[16] Univ Southern Denmark, Clin Inst, Odense, Denmark

[17] Univ Hosp Padua, Dept Syst Med, Padua, Italy

[18] Penn State Univ, Dept Med & Pediat, Allergy Asthma & Immunol Div, Hershey, PA USA

[19] Semmelweis Univ, Hungarian Angioedema Ctr Reference & Excellence, Dept Internal Med & Haematol, Budapest, Hungary

[20] Clin City Hosp 52, Urticaria Ctr Reference & Excellence, Moscow Res & Clin Ctr Allergy & Immunol, Moscow, Russia

[21] Sechenov Univ, Dept Clin Immunol & Allergol, Moscow, Russia

[22] Astana Med Univ, Dept Pulmonol, Astana, Kazakhstan

[23] Hosp Univ Vall dHebron, Allergy Sect Dept, Dept Internal Med, Barcelona, Spain

[24] Univ Autonoma Barcelona, Inst Recerca Vall dHebron, Allergy Dept, Allergy Res Unit, Barcelona, Spain

[25] North Bristol NHS Trust, Clin Immunol, Bristol, England

[26] Inst Politecn Nacl SEPI ENMH, Dept Immunol, Mexico City, Mexico

[27] Hiroshima City Hiroshima Citizens Hosp, Dept Dermatol, Hiroshima, Japan

[28] Royal Free London NHS Fdn Trust, London, England

[29] Med Univ Vienna, Dept Dermatol, Vienna, Austria

[30] Takeda Pharmaceut, Hoofddorp, Netherlands

[31] Hosp Univ Bellvitge, Inst Recerca IDIBELL LHospitalet Llobregat, Allergol Dept, Barcelona, Spain

[32] Univ Auckland, Dept Immunol, Auckland Dist Hlth Board, Auckland, New Zealand

[33] Univ Auckland, Dept Med, Auckland, New Zealand

[34] Univ Texas Hlth Sci Ctr, Allergy Div, Internal Med, Dallas, TX USA

[35] Asma Inmunol Clin, Unidad Alergia, Buenos Aires, Argentina

[36] Vilnius Univ, Clin Chest Dis Immunol & Allergol, Dept Pulmonol & Allergol, Med Fac,VUH Santaros Klin, Vilnius, Lithuania

[37] UMAE Hosp Especial CMNSXXI, Allergy & Clin Immunol Dept, IMSS, Mexico City, Mexico

[38] Pharvaris GmbH, Head Evidence & Outcomes Res, Zug, Switzerland

[39] Natl Inst Pediat, Genet Unit Nutr, Mexico City, Mexico

[40] Univ Cape Town, Groote Schuur Hosp, Div Allergy & Clin Immunol, Cape Town, South Africa

[41] Univ Cape, Allergy & Immunol Unit, Town Lung Inst, Cape Town, South Africa

[42] Jagiellonian Univ, Dept Clin & Environm Allergol, Med Coll, Krakow, Poland

[43] Barzilai Univ, Angioedema Ctr, Med Ctr, Ashqelon, Israel

[44] Univ Calif San Diego, Div Rheumatol Allergy & Immunol, La Jolla, CA USA

[45] Med Univ Sofia, Dept Allergol, Sofia, Bulgaria

[46] Univ Hosp Alexandrovska, Angioedema Ctr Reference & Excellence Bulgaria, Sofia, Bulgaria

[47] McMaster Univ, Dept Med, Hamilton, ON, Canada

来源：

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY-IN PRACTICE | 2024年 / 12卷 / 06期

关键词：

Acute treatment; Consensus; Core outcome set; Delphi; Hereditary angioedema; Randomized controlled trial; Outcome; VALIDATION;

D O I：

10.1016/j.jaip.2024.04.007

中图分类号：

R392 [医学免疫学];

学科分类号：

100102 ;

摘要：

BACKGROUND: Clinical trials investigating drugs for the acute treatment of hereditary angioedema attacks have assessed many different outcomes. This heterogeneity limits the comparability of trial results and may lead to selective outcome reporting bias and a high burden on trial participants. OBJECTIVE: To achieve consensus on a core outcome set composed of key outcomes that ideally should be used in all clinical efficacy trials involving the acute treatment of hereditary angioedema attacks. METHODS: We conducted a Delphi consensus study involving all relevant parties: patients with hereditary angioedema, hereditary angioedema expert clinicians and clinical researchers, pharmaceutical companies, and regulatory bodies. Two Internetbased survey rounds were conducted. In round 1, panelists indicated the importance of individual outcomes used in clinical trials on a 9 -point Likert scale. Based on these results, a core outcome set was developed and voted on by panelists in round 2. RESULTS: A total of 58 worldwide panelists completed both rounds. The fi rst round demonstrated high importance scores and substantial agreement among the panelists. In the second round, a consensus of 90% or greater was achieved on a core outcome set consisting of fi ve key outcomes: change in overall symptom severity at one predetermined time point between 15 minutes and 4 hours after treatment, time to end of progression of all symptoms, the need for rescue medication during the entire attack, impairment of daily activities, and treatment satisfaction. CONCLUSIONS: This international study obtained a high level of consensus on a core outcome set for the acute treatment of hereditary angioedema attacks, consisting of fi ve key outcomes. Crown Copyright (c) 2024 Published by Elsevier Inc. on behalf of the American Academy of Allergy, Asthma & Immunology. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/). (J Allergy Clin Immunol Pract 2024;12:1614-21)

引用

页码：1614 / 1621

页数：8

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