Factors Influencing Placebo Responses in Rheumatoid Arthritis Clinical Trials: A Meta-Analysis of Randomized, Double-Blind, Placebo-Controlled Studies.


Journal

Clinical drug investigation
ISSN: 1179-1918
Titre abrégé: Clin Drug Investig
Pays: New Zealand
ID NLM: 9504817

Informations de publication

Date de publication:
Mar 2020
Historique:
pubmed: 19 1 2020
medline: 28 7 2020
entrez: 19 1 2020
Statut: ppublish

Résumé

A better understanding of placebo responses and the specific factors influencing these outcomes is important for clinical trial design. We investigated the magnitude of placebo responses at 3 months and the potential factors influencing these outcomes in rheumatoid arthritis (RA) clinical trials. We conducted a systematic review of randomized placebo-controlled trials of pharmacological agents for RA identified from PubMed, Embase, and Cochrane Central Register of Controlled Trials databases. The primary placebo outcome was American College of Rheumatology 20% response rate (ACR20). Data were pooled with a random-effects model. Factors influencing placebo response were assessed by meta-regression analyses. Subgroup analyses were performed for studies conducted in non-Western countries only versus in Western countries (North America and/or Europe) only or both. The meta-analysis included 88 studies comprising 8406 patients receiving a placebo. The pooled estimate of placebo ACR20 was 29.0% (range 10.0-46.2; 95% confidence interval 27.2-30.9). Placebo ACR20 was negatively associated with trials in non-Western (Asian) countries and patient populations showing an inadequate response to biological disease-modifying antirheumatic drugs (DMARDs) in the multivariable analysis, whereas it was positively associated with the year of publication. No background DMARD treatment was also a negative predictor (albeit statistically non-significant). In subgroup analyses of Western and multiregional studies, study population and publication year were significant factors. Our meta-analysis suggests that study location, patient population, and a background DMARD treatment influence placebo ACR20. These along with placebo response temporal profiles have important implications for designing and interpreting RA clinical trials.

Sections du résumé

BACKGROUND AND OBJECTIVE OBJECTIVE
A better understanding of placebo responses and the specific factors influencing these outcomes is important for clinical trial design. We investigated the magnitude of placebo responses at 3 months and the potential factors influencing these outcomes in rheumatoid arthritis (RA) clinical trials.
METHODS METHODS
We conducted a systematic review of randomized placebo-controlled trials of pharmacological agents for RA identified from PubMed, Embase, and Cochrane Central Register of Controlled Trials databases. The primary placebo outcome was American College of Rheumatology 20% response rate (ACR20). Data were pooled with a random-effects model. Factors influencing placebo response were assessed by meta-regression analyses. Subgroup analyses were performed for studies conducted in non-Western countries only versus in Western countries (North America and/or Europe) only or both.
RESULTS RESULTS
The meta-analysis included 88 studies comprising 8406 patients receiving a placebo. The pooled estimate of placebo ACR20 was 29.0% (range 10.0-46.2; 95% confidence interval 27.2-30.9). Placebo ACR20 was negatively associated with trials in non-Western (Asian) countries and patient populations showing an inadequate response to biological disease-modifying antirheumatic drugs (DMARDs) in the multivariable analysis, whereas it was positively associated with the year of publication. No background DMARD treatment was also a negative predictor (albeit statistically non-significant). In subgroup analyses of Western and multiregional studies, study population and publication year were significant factors.
CONCLUSIONS CONCLUSIONS
Our meta-analysis suggests that study location, patient population, and a background DMARD treatment influence placebo ACR20. These along with placebo response temporal profiles have important implications for designing and interpreting RA clinical trials.

Identifiants

pubmed: 31953723
doi: 10.1007/s40261-020-00887-6
pii: 10.1007/s40261-020-00887-6
doi:

Substances chimiques

Antirheumatic Agents 0

Types de publication

Journal Article Meta-Analysis Systematic Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

197-209

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Auteurs

Kota Nagai (K)

Department of Pharmacoepidemiology, Graduate School of Medicine and Public Health, Kyoto University, Yoshida-Konoe-cho, Sakyo-ku, Kyoto, 606-8501, Japan.
Eisai Co., Ltd., 4-6-10 Koishikawa, Bunkyo-ku, Tokyo, 112-8088, Japan.

Keisuke Matsubayashi (K)

Department of Pharmacoepidemiology, Graduate School of Medicine and Public Health, Kyoto University, Yoshida-Konoe-cho, Sakyo-ku, Kyoto, 606-8501, Japan.

Kazuki Ide (K)

Department of Pharmacoepidemiology, Graduate School of Medicine and Public Health, Kyoto University, Yoshida-Konoe-cho, Sakyo-ku, Kyoto, 606-8501, Japan.
Center for the Promotion of Interdisciplinary Education and Research, Kyoto University, Kyoto, 606-8501, Japan.

Kahori Seto (K)

Department of Pharmacoepidemiology, Graduate School of Medicine and Public Health, Kyoto University, Yoshida-Konoe-cho, Sakyo-ku, Kyoto, 606-8501, Japan.

Yohei Kawasaki (Y)

Biostatistics Section, Clinical Research Center, Chiba University Hospital, 1-8-1 Inohana, Chuo-ku, Chiba-shi, Chiba, 260-8677, Japan.

Koji Kawakami (K)

Department of Pharmacoepidemiology, Graduate School of Medicine and Public Health, Kyoto University, Yoshida-Konoe-cho, Sakyo-ku, Kyoto, 606-8501, Japan. kawakami.koji.4e@kyoto-u.ac.jp.
Center for the Promotion of Interdisciplinary Education and Research, Kyoto University, Kyoto, 606-8501, Japan. kawakami.koji.4e@kyoto-u.ac.jp.

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Classifications MeSH