A core outcome set for efficacy of acute treatment of hereditary angioedema
Copyright © 2024. Published by Elsevier Inc..
BACKGROUND: Clinical trials investigating drugs for acute treatment of hereditary angioedema attacks have assessed many different outcomes. This heterogeneity limits 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 comprising key outcomes that should ideally be utilized in all clinical efficacy trials involving acute treatment of hereditary angioedema attacks.
METHODS: A Delphi consensus study was conducted involving all relevant parties: hereditary angioedema patients, hereditary angioedema expert clinicians and clinical researchers, pharmaceutical companies, and regulatory bodies. Two internet-based 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: Fifty-eight worldwide panelists completed both rounds. The first round demonstrated high importance scores and substantial agreement among the panelists. In the second round, a consensus of ≥90% was achieved on a core outcome set consisting of five 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, need for rescue medication during the entire attack, impairment of daily activities, and treatment satisfaction.
CONCLUSION: This international study obtained a high level of consensus on a core outcome set for acute treatment of hereditary angioedema attacks consisting of five key outcomes.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2024 |
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| Erschienen: |
2024 |
| Enthalten in: |
Zur Gesamtaufnahme - year:2024 |
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| Enthalten in: |
The journal of allergy and clinical immunology. In practice - (2024) vom: 10. Apr. |
| Sprache: |
Englisch |
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| Links: |
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| Themen: |
Acute treatment |
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| Anmerkungen: |
Date Revised 12.04.2024 published: Print-Electronic Citation Status Publisher |
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| doi: |
10.1016/j.jaip.2024.04.007 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
NLM370983335 |
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| 100 | 1 | |a Petersen, Remy S |e verfasserin |4 aut | |
| 245 | 1 | 2 | |a A core outcome set for efficacy of acute treatment of hereditary angioedema |
| 264 | 1 | |c 2024 | |
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| 500 | |a Date Revised 12.04.2024 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status Publisher | ||
| 520 | |a Copyright © 2024. Published by Elsevier Inc. | ||
| 520 | |a BACKGROUND: Clinical trials investigating drugs for acute treatment of hereditary angioedema attacks have assessed many different outcomes. This heterogeneity limits comparability of trial results and may lead to selective outcome reporting bias and a high burden on trial participants | ||
| 520 | |a OBJECTIVE: To achieve consensus on a Core Outcome Set comprising key outcomes that should ideally be utilized in all clinical efficacy trials involving acute treatment of hereditary angioedema attacks | ||
| 520 | |a METHODS: A Delphi consensus study was conducted involving all relevant parties: hereditary angioedema patients, hereditary angioedema expert clinicians and clinical researchers, pharmaceutical companies, and regulatory bodies. Two internet-based 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 | ||
| 520 | |a RESULTS: Fifty-eight worldwide panelists completed both rounds. The first round demonstrated high importance scores and substantial agreement among the panelists. In the second round, a consensus of ≥90% was achieved on a core outcome set consisting of five 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, need for rescue medication during the entire attack, impairment of daily activities, and treatment satisfaction | ||
| 520 | |a CONCLUSION: This international study obtained a high level of consensus on a core outcome set for acute treatment of hereditary angioedema attacks consisting of five key outcomes | ||
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| 650 | 4 | |a Delphi | |
| 650 | 4 | |a consensus | |
| 650 | 4 | |a core outcome set | |
| 650 | 4 | |a hereditary angioedema | |
| 650 | 4 | |a outcome | |
| 650 | 4 | |a randomized controlled trial | |
| 700 | 1 | |a Fijen, Lauré M |e verfasserin |4 aut | |
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