Protocol for the development of a core outcome set for clinical trials in primary sclerosing cholangitis
Standard
Protocol for the development of a core outcome set for clinical trials in primary sclerosing cholangitis. / Hussain, Nasir; Ma, Christopher; Hirschfield, Gideon; Walmsley, Martine; Hanford, Paula; Vesterhus, Mette; Kowdley, Kris; Bergquist, Annika; Ponsioen, Cyriel; Levy, Cynthia; Assis, David; Schramm, Christoph; Bowlus, Christopher; Trauner, Michael; Aiyegbusi, Olalekan Lee; Jairath, Vipul; Trivedi, Palak J.
In: BMJ OPEN, Vol. 14, No. 6, 26.06.2024, p. e080143.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
Harvard
APA
Vancouver
Bibtex
}
RIS
TY - JOUR
T1 - Protocol for the development of a core outcome set for clinical trials in primary sclerosing cholangitis
AU - Hussain, Nasir
AU - Ma, Christopher
AU - Hirschfield, Gideon
AU - Walmsley, Martine
AU - Hanford, Paula
AU - Vesterhus, Mette
AU - Kowdley, Kris
AU - Bergquist, Annika
AU - Ponsioen, Cyriel
AU - Levy, Cynthia
AU - Assis, David
AU - Schramm, Christoph
AU - Bowlus, Christopher
AU - Trauner, Michael
AU - Aiyegbusi, Olalekan Lee
AU - Jairath, Vipul
AU - Trivedi, Palak J
N1 - © Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.
PY - 2024/6/26
Y1 - 2024/6/26
N2 - BACKGROUND: Primary sclerosing cholangitis (PSC) is a progressive immune-mediated liver disease, for which no medical therapy has been shown to slow disease progression. However, the horizon for new therapies is encouraging, with several innovative clinical trials in progress. Despite these advancements, there is considerable heterogeneity in the outcomes studied, with lack of consensus as to what outcomes to measure, when to measure and how to measure. Furthermore, there has been a paradigm shift in PSC treatment targets over recent years, moving from biochemistry-based endpoints to histological assessment of liver fibrosis, imaging-based biomarkers and patient-reported outcome measures. The abundance of new interventional trials and evolving endpoints pose opportunities for all stakeholders involved in evaluating novel therapies. To this effect, there is a need to harmonise measures used in clinical trials through the development of a core outcome set (COS).METHODS AND ANALYSIS: Synthesis of a PSC-specific COS will be conducted in four stages. Initially, a systematic literature review will be performed to identify outcomes previously used in PSC trials, followed by semistructured qualitative interviews conducted with key stakeholders. The latter may include patients, clinicians, researchers, pharmaceutical industry representatives and healthcare payers and regulatory agencies, to identify additional outcomes of importance. Using the outcomes generated from the literature review and stakeholder interviews, an international two-round Delphi survey will be conducted to prioritise outcomes for inclusion in the COS. Finally, a consensus meeting will be convened to ratify the COS and disseminate findings for application in future PSC trials.ETHICS AND DISSEMINATION: Ethical approval has been granted by the East Midlands-Leicester Central Research Ethics Committee (Ref: 24/EM/0126) for this study. The COS from this study will be widely disseminated including publication in peer-reviewed journals, international conferences, promotion through patient-support groups and made available on the Core Outcomes Measurement in Effectiveness Trials (COMET) database.TRIAL REGISTRATION NUMBER: 1239.
AB - BACKGROUND: Primary sclerosing cholangitis (PSC) is a progressive immune-mediated liver disease, for which no medical therapy has been shown to slow disease progression. However, the horizon for new therapies is encouraging, with several innovative clinical trials in progress. Despite these advancements, there is considerable heterogeneity in the outcomes studied, with lack of consensus as to what outcomes to measure, when to measure and how to measure. Furthermore, there has been a paradigm shift in PSC treatment targets over recent years, moving from biochemistry-based endpoints to histological assessment of liver fibrosis, imaging-based biomarkers and patient-reported outcome measures. The abundance of new interventional trials and evolving endpoints pose opportunities for all stakeholders involved in evaluating novel therapies. To this effect, there is a need to harmonise measures used in clinical trials through the development of a core outcome set (COS).METHODS AND ANALYSIS: Synthesis of a PSC-specific COS will be conducted in four stages. Initially, a systematic literature review will be performed to identify outcomes previously used in PSC trials, followed by semistructured qualitative interviews conducted with key stakeholders. The latter may include patients, clinicians, researchers, pharmaceutical industry representatives and healthcare payers and regulatory agencies, to identify additional outcomes of importance. Using the outcomes generated from the literature review and stakeholder interviews, an international two-round Delphi survey will be conducted to prioritise outcomes for inclusion in the COS. Finally, a consensus meeting will be convened to ratify the COS and disseminate findings for application in future PSC trials.ETHICS AND DISSEMINATION: Ethical approval has been granted by the East Midlands-Leicester Central Research Ethics Committee (Ref: 24/EM/0126) for this study. The COS from this study will be widely disseminated including publication in peer-reviewed journals, international conferences, promotion through patient-support groups and made available on the Core Outcomes Measurement in Effectiveness Trials (COMET) database.TRIAL REGISTRATION NUMBER: 1239.
KW - Humans
KW - Cholangitis, Sclerosing/therapy
KW - Research Design
KW - Clinical Trials as Topic
KW - Delphi Technique
KW - Outcome Assessment, Health Care
KW - Endpoint Determination
KW - Systematic Reviews as Topic
U2 - 10.1136/bmjopen-2023-080143
DO - 10.1136/bmjopen-2023-080143
M3 - SCORING: Journal article
C2 - 38926149
VL - 14
SP - e080143
JO - BMJ OPEN
JF - BMJ OPEN
SN - 2044-6055
IS - 6
ER -