Botulinum neurotoxin treatment in jerky and tremulous functional movement disorders: a double-blind, randomised placebo-controlled trial with an open-label extension. Issue 11 (20th June 2019)

• Record Type: Journal Article
• Authors: Dreissen, Yasmine Emma Maria; Dijk, Joke M; Gelauff, Jeannette M; Zoons, Evelien; van Poppelen, Daniël; Contarino, Maria Fiorella; Zutt, Rodi; Post, Bart; Munts, Alexander G; Speelman, Johannes D; Cath, Danielle C; de Haan, Rob J; Koelman, Johannes HTM; Tijssen, Marina A J
• Is Part Of: Journal of neurology, neurosurgery and psychiatry. Volume 90:Issue 11(2019)
• Page Start: 1244
• Page End: 1250
• Publication Date: 2019-06-20
• Subjects: Neurology -- Periodicals; Nervous system -- Surgery -- Periodicals; Psychiatry -- Periodicals; 616.8
• DOI: 10.1136/jnnp-2018-320071
• Languages: English
• ISSNs: 0022-3050

Abstract

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