Botulinum neurotoxin treatment in jerky and tremulous functional movement disorders: a double-blind, randomised placebo-controlled trial with an open-label extensionDreissen, Y. E. M., Dijk, J. M., Gelauff, J. M., Zoons, E., van Poppelen, D., Contarino, M. F., Zutt, R., Post, B., Munts, A. G., Speelman, J. D., Cath, D. C., de Haan, R. J., Koelman, J. H. T. M. & Tijssen, M. A. J., Nov-2019, In : JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY. 90, 11, p. 1244-1250 7 p.
Research output: Contribution to journal › Article › Academic › peer-review
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.
|Number of pages||7|
|Journal||JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY|
|Publication status||Published - Nov-2019|
- SYMPTOMS, CRITERIA, TOXIN