Development and Application of the Placebo Response Model in Clinical Trials for Primary Sjögren’s Syndrome

This study aimed to develop a placebo response model for pharmaceutical clinical trials of primary Sjogren’s syndrome,and to quantitatively analyze the distribution and related factors influencing the placebo response to further optimize the design of clinical trials and evaluate the results of sing...

Descripción completa

Guardado en:

Detalles Bibliográficos
Autores principales:	ZHI-Zhou Wang, Qing-Shan Zheng, Hong-Xia Liu, Lu-Jin Li
Formato:	article
Lenguaje:	EN
Publicado:	Frontiers Media S.A. 2021
Materias:	Sjögren’s syndrome placebo response EULAR Sjögren’s syndrome disease activity index (ESSDAI) Model-based meta-analysis simulation Immunologic diseases. Allergy RC581-607
Acceso en línea:	https://doaj.org/article/659a180008d140a49543b119663a340b
Etiquetas:	Agregar Etiqueta Sin Etiquetas, Sea el primero en etiquetar este registro!

id	oai:doaj.org-article:659a180008d140a49543b119663a340b
record_format	dspace
spelling	oai:doaj.org-article:659a180008d140a49543b119663a340b2021-11-16T06:03:54ZDevelopment and Application of the Placebo Response Model in Clinical Trials for Primary Sjögren’s Syndrome1664-322410.3389/fimmu.2021.783246https://doaj.org/article/659a180008d140a49543b119663a340b2021-11-01T00:00:00Zhttps://www.frontiersin.org/articles/10.3389/fimmu.2021.783246/fullhttps://doaj.org/toc/1664-3224This study aimed to develop a placebo response model for pharmaceutical clinical trials of primary Sjogren’s syndrome,and to quantitatively analyze the distribution and related factors influencing the placebo response to further optimize the design of clinical trials and evaluate the results of single-arm clinical trials. Public databases, including PubMed, Embase, and Cochrane Library were searched for reports on randomized placebo-controlled trials for Sjögren’s syndrome which used the change from baseline in ESSDAI score as the primary outcome. The model-based meta-analysis method was used to evaluate the time course and the related influencing factors of the placebo response for ESSDAI in such clinical trials. A virtual placebo control group was constructed based on the final placebo response model to determine the treatment efficacy of belimumab and cyclosporine A for primary Sjögren’s syndrome in a single-arm study. A total of 12 studies involving 450 subjects were included in the analysis. The established model described the time-course characteristics of the changes in ESSDAI score from the baseline in the 48 weeks placebo group. We found that the onset time of placebo response was approximately 12 weeks, and its efficacy plateaued at 48 weeks. The baseline ESSDAI score had a significant effect on the maximum value of the placebo response; the maximum value of the placebo response decreased by 0.552 for every 1 score rise in the baseline ESSDAI score. The efficacy of belimumab and cyclosporine A in the single-arm trial was comparable to that of the placebo response at the same baseline; no significant therapeutic advantage was observed. The placebo response model established in this study could provide a basis for designing clinical trials for primary Sjogren’s syndrome in the future. It may also provide a reliable external efficacy control standard for single-arm clinical trials.ZHI-Zhou WangQing-Shan ZhengHong-Xia LiuLu-Jin LiFrontiers Media S.A.articleSjögren’s syndromeplacebo responseEULAR Sjögren’s syndrome disease activity index (ESSDAI)Model-based meta-analysissimulationImmunologic diseases. AllergyRC581-607ENFrontiers in Immunology, Vol 12 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Sjögren’s syndrome placebo response EULAR Sjögren’s syndrome disease activity index (ESSDAI) Model-based meta-analysis simulation Immunologic diseases. Allergy RC581-607
spellingShingle	Sjögren’s syndrome placebo response EULAR Sjögren’s syndrome disease activity index (ESSDAI) Model-based meta-analysis simulation Immunologic diseases. Allergy RC581-607 ZHI-Zhou Wang Qing-Shan Zheng Hong-Xia Liu Lu-Jin Li Development and Application of the Placebo Response Model in Clinical Trials for Primary Sjögren’s Syndrome
description	This study aimed to develop a placebo response model for pharmaceutical clinical trials of primary Sjogren’s syndrome,and to quantitatively analyze the distribution and related factors influencing the placebo response to further optimize the design of clinical trials and evaluate the results of single-arm clinical trials. Public databases, including PubMed, Embase, and Cochrane Library were searched for reports on randomized placebo-controlled trials for Sjögren’s syndrome which used the change from baseline in ESSDAI score as the primary outcome. The model-based meta-analysis method was used to evaluate the time course and the related influencing factors of the placebo response for ESSDAI in such clinical trials. A virtual placebo control group was constructed based on the final placebo response model to determine the treatment efficacy of belimumab and cyclosporine A for primary Sjögren’s syndrome in a single-arm study. A total of 12 studies involving 450 subjects were included in the analysis. The established model described the time-course characteristics of the changes in ESSDAI score from the baseline in the 48 weeks placebo group. We found that the onset time of placebo response was approximately 12 weeks, and its efficacy plateaued at 48 weeks. The baseline ESSDAI score had a significant effect on the maximum value of the placebo response; the maximum value of the placebo response decreased by 0.552 for every 1 score rise in the baseline ESSDAI score. The efficacy of belimumab and cyclosporine A in the single-arm trial was comparable to that of the placebo response at the same baseline; no significant therapeutic advantage was observed. The placebo response model established in this study could provide a basis for designing clinical trials for primary Sjogren’s syndrome in the future. It may also provide a reliable external efficacy control standard for single-arm clinical trials.
format	article
author	ZHI-Zhou Wang Qing-Shan Zheng Hong-Xia Liu Lu-Jin Li
author_facet	ZHI-Zhou Wang Qing-Shan Zheng Hong-Xia Liu Lu-Jin Li
author_sort	ZHI-Zhou Wang
title	Development and Application of the Placebo Response Model in Clinical Trials for Primary Sjögren’s Syndrome
title_short	Development and Application of the Placebo Response Model in Clinical Trials for Primary Sjögren’s Syndrome
title_full	Development and Application of the Placebo Response Model in Clinical Trials for Primary Sjögren’s Syndrome
title_fullStr	Development and Application of the Placebo Response Model in Clinical Trials for Primary Sjögren’s Syndrome
title_full_unstemmed	Development and Application of the Placebo Response Model in Clinical Trials for Primary Sjögren’s Syndrome
title_sort	development and application of the placebo response model in clinical trials for primary sjögren’s syndrome
publisher	Frontiers Media S.A.
publishDate	2021
url	https://doaj.org/article/659a180008d140a49543b119663a340b
work_keys_str_mv	AT zhizhouwang developmentandapplicationoftheplaceboresponsemodelinclinicaltrialsforprimarysjogrenssyndrome AT qingshanzheng developmentandapplicationoftheplaceboresponsemodelinclinicaltrialsforprimarysjogrenssyndrome AT hongxialiu developmentandapplicationoftheplaceboresponsemodelinclinicaltrialsforprimarysjogrenssyndrome AT lujinli developmentandapplicationoftheplaceboresponsemodelinclinicaltrialsforprimarysjogrenssyndrome
_version_	1718426669440565248

Development and Application of the Placebo Response Model in Clinical Trials for Primary Sjögren’s Syndrome

Ejemplares similares