Faiza Shafiq1, Dimos-Dimitrios Mitsikostas2, Panagiotis Zis1,3. 1. a Department of Neurology , Sheffield Teaching Hospitals NHS Trust , Sheffield , UK. 2. b 1st Department of Neurology , National and Kapodistrian University of Athens , Athens , Greece , and. 3. c Academic Directorate of Neurosciences , Sheffield Teaching Hospitals NHS Trust , Sheffield , UK.
Abstract
INTRODUCTION: Nocebo is very prevalent among neurological disorders, resulting in low adherence and treatment outcome. We sought to examine the AEs following placebo administration in placebo-controlled randomised clinical trials (RCTs) for motor neuron disease (MND). METHODS: After a systematic literature search for RCTs for MND pharmacotherapy treatments, we assessed the number of discontinuations because of placebo intolerance. RESULTS: Data were extracted from 12 RCTs fulfilling our search criteria. Approximately eight in 10 placebo-treated patients (78.3%, 95% CI 74.3-82.0%) reported at least one AE and approximately one in 12 placebo-treated patients discontinued placebo treatment because of AEs (8.4%, 95% CI 6.7-10.4%). All patients participating in the MND trials reported similar AEs independently of the study arm to which they belonged. CONCLUSION: Our study indicates significant nocebo in trials for MND treatment, adversely affecting adherence and efficacy of current treatments in clinical practice, with additional implications for trial design.
INTRODUCTION: Nocebo is very prevalent among neurological disorders, resulting in low adherence and treatment outcome. We sought to examine the AEs following placebo administration in placebo-controlled randomised clinical trials (RCTs) for motor neuron disease (MND). METHODS: After a systematic literature search for RCTs for MND pharmacotherapy treatments, we assessed the number of discontinuations because of placebo intolerance. RESULTS: Data were extracted from 12 RCTs fulfilling our search criteria. Approximately eight in 10 placebo-treated patients (78.3%, 95% CI 74.3-82.0%) reported at least one AE and approximately one in 12 placebo-treated patients discontinued placebo treatment because of AEs (8.4%, 95% CI 6.7-10.4%). All patients participating in the MND trials reported similar AEs independently of the study arm to which they belonged. CONCLUSION: Our study indicates significant nocebo in trials for MND treatment, adversely affecting adherence and efficacy of current treatments in clinical practice, with additional implications for trial design.
Entities:
Keywords:
Nocebo; adverse events; motor neuron disease; placebo; trial design