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J Cyst Fibros. 2019 Feb 13. pii: S1569-1993(18)30833-6. doi: 10.1016/j.jcf.2019.02.003. [Epub ahead of print]

Do patients with cystic fibrosis participating in clinical trials demonstrate placebo response? A meta-analysis.

Author information

1
UMR 5558 CNRS, Equipe EMET, Université Claude Bernard Lyon 1, Lyon, France; Centre de ressources et de compétences de la mucoviscidose, Hospices Civils de Lyon, Lyon, France.
2
UMR 5558 CNRS, Equipe EMET, Université Claude Bernard Lyon 1, Lyon, France.
3
Meta-Research Innovation Center at Stanford (METRICS), Stanford University, Stanford, California.
4
Department of Clinical Pharmacology, Université Claude Bernard Lyon 1, Lyon, France.
5
UMR 5558 CNRS, Equipe EMET, Université Claude Bernard Lyon 1, Lyon, France; EPICIME-Clinical Investigation Center, INSERM CIC1407/UMR5558, Bron, France.
6
UMR 5558 CNRS, Equipe EMET, Université Claude Bernard Lyon 1, Lyon, France; Centre de ressources et de compétences de la mucoviscidose, Hospices Civils de Lyon, Lyon, France. Electronic address: philippe.reix@chu-lyon.fr.

Abstract

BACKGROUND:

Patients' and families' expectation that a cure for cystic fibrosis (CF) will be found is high. In other debilitating conditions, high expectation has been shown to drive a strong placebo response (PR). Therefore, our goal was to evaluate PR on objective continuous outcomes (FEV1, BMI) and the CF Questionnaire Revised-Respiratory Domain (CFQR-RD) monitored during randomised clinical trials (RCTs) for CF.

METHODS:

We conducted a meta-analysis after a systematic review of the literature carried out to identify RCTs with FEV1, CFQR-RD and BMI as outcome measures. The standardised mean difference (SMD) was calculated to estimate the PR. A meta-regression analysis was conducted to assess other contributing factors on PR such as study design, trial duration, patient age and disease severity.

RESULTS:

Out of 289 RCTs found in the search, we identified 61 articles (published from 1987 to 2017) with respectively 59, 17 and 9 reporting FEV1, CFQR-RD and BMI at the start and at the end of the RCTs. No significant PR was found on FEV1 or CFQR-RD. However, a small but significant PR was found on BMI SMD, 0.09 (95% CI (0.01; 0.17); p = 0.03).

CONCLUSION:

The PR seems higher when measuring BMI. However, it is not clear whether this improvement can be explained by a PR alone.

KEYWORDS:

Clinical trials; Cystic fibrosis; Meta-analysis; Placebo response; Randomised

PMID:
30772244
DOI:
10.1016/j.jcf.2019.02.003

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