Botulinum neurotoxin treatment in jerky and tremulous functional movement disorders: a double-blind, randomised placebo-controlled trial with an open-label extension

摘要: Objective To study the effect of botulinum neurotoxin (BoNT) treatment in jerky and tremulous functional movement disorders (FMD). Methods Patients with invalidating, chronic (>1 year) symptoms were randomly assigned to two subsequent treatments BoNT or placebo every 3 months stratification according symptom localisation. Improvement on dichotomised Clinical Global Impression-Improvement scale (CGI-I) (improvement vs no change worsening) at 4 months, assessed by investigators blinded allocated was primary outcome. Subsequently all patients treated a ten month open-label phase. Results Between January 2011 February 2015 total 239 screened for eligibility whom 48 included. No difference found outcome (BoNT 16 25 (64.0%) Placebo 13 23 (56.5%); proportional 0.075 (95% CI −0.189 0.327; p=0.77). Secondary outcomes (symptom severity, disease burden, disability, quality life psychiatric symptoms) showed between-group differences. The phase improvement CGI-I 19/43 (44.2%) remaining patients, 35/43 (81.4%) compared baseline. Conclusions In this double-blind randomised controlled trial FMD, we evidence improved placebo. Motor large proportion both groups which sustained This underlines substantial potential FMD recover may stimulate further exploration placebo-therapies these patients. Trial registration number NTR2478