Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2021 Mar;268(3):903-912.
doi: 10.1007/s00415-020-10217-7. Epub 2020 Sep 16.

Patient perspectives on the therapeutic profile of botulinum neurotoxin type A in cervical dystonia

Cynthia Comella  1 , Joaquim J Ferreira  2   3 , Emilie Pain  4 , Marion Azoulai  4 , Savary Om  5
Affiliations

Patient perspectives on the therapeutic profile of botulinum neurotoxin type A in cervical dystonia

Cynthia Comella et al. J Neurol. 2021 Mar.

Erratum in

Abstract

Background: Botulinum neurotoxin type A (BoNT-A) is an effective pharmacological treatment for the management of cervical dystonia (CD) that requires repeated administration at variable intervals. We explored patient perceptions of the impact of CD and the waning of BoNT-A therapeutic effects.

Methods: An internet-based survey was conducted through Carenity, a global online patient community, from May to September 2019. Eligible respondents were adults with CD who had ≥ 2 previous BoNT-A injections.

Results: 209 respondents (81% females; mean age of 49.7 years) met the screening criteria. The mean BoNT-A injection frequency was 3.9 injections/year. The mean reported onset of BoNT-A therapeutic effect was 11.7 days and the time to peak effect was 4.5 weeks. Symptom re-emergence between injections was common (88%); the time from injection to symptom re-emergence was 73.6 days (~ 10.5 weeks). Treatment was not reported to completely abolish symptoms, even at peak effect. However, symptom severity was rated (0 = no symptoms; 10 = very strong symptoms) as lowest at the peak of treatment effects (mean scores ~ 3/10), increasing as the effects of treatment start waning (~ 5.5/10) and was strongest one day before the next session (~ 7-8/10). The impact of CD on quality of life followed the same 'rollercoaster' pattern.

Conclusions: This survey highlights the burden of CD symptoms, even in patients undergoing regular treatment. Symptom re-emergence is common and has significant impact on daily activities and quality of life. Greater awareness of the therapeutic profile of BoNT-A treatment should lead to better informed therapeutic discussions and planning.

Keywords: Botulinum toxin; Cervical dystonia; Patient; Survey; Treatment; Waning of effect.

PubMed Disclaimer

Conflict of interest statement

Dr. Comella serves on the editorial board of Clinical Neuropharmacology and Sleep Medicine. She receives compensation/honoraria for services as a consultant or an advisory committee member: Acorda Therapeutics, Allergan, Inc; Lundbeck Ltd.; Merz Pharmaceuticals; Acadia Pharmaceuticals; Ipsen Pharmaceuticals, Jazz. Pharmaceuticals, Neurocrine Biosciences Inc., Revance Therapeutic, Sunovion., AEON Biopharma. She receives royalties from Cambridge University Press and Wolters Kluwer. She receives research support from the Parkinson’s Disease Foundation. Joaquim J. Ferreira has held consultancy functions with GlaxoSmithKline, Novartis, TEVA, Lundbeck, Solvay, Abbott, Abbvie, BIAL, Merck-Serono, Merz, Ipsen, Biogen, NeuroDerm, Zambon, Sunovion, Affiris, ONO; has received lecture fees from Biogen and BIAL, Sunovion, ONO, Zambon, Abbvie; has received grants from GlaxoSmithKline, Grunenthal, MSD, Allergan, Novartis, Fundação MSD (Portugal), Medtronic and Teva; has been employed by Faculdade de Medicina de Lisboa and CNS—Campus Neurológico Sénior. Marion Azoulai and Emilie Pain are employed by Carenity who received funding from Ipsen for conducting this survey. Savary Om was an Ipsen employee at the time of study.

Figures

Fig. 1
Fig. 1
Current therapy for CD symptoms. Question: Which treatments are you currently receiving for your CD? (N = 209). *Oral medications such as muscle relaxant or baclofen. BoNT-A botulinum neurotoxin type A, CD cervical dystonia
Fig. 2
Fig. 2
Patient experiences of a BoNT-A response b CD symptom intensity and c impact on quality of life across a single injection cycle. a Schematic representing the mean [95% CI] time to onset and peak therapeutic effects and mean time to symptom re-emergence. Respondents were asked: On average, how many days or weeks after your BoNT-A injections do you experience (1) The first effect of the treatment on your cervical dystonia (2) the maximum effects of the treatment on your CD (in days or weeks). In general, how long after your BoNT-A injections do your pre-existing symptoms begin to reappear? N = 209 respondents. *Respondents indicated the time between the last 2 BoNT-A sessions. Figures b and c: Schematics representing mean symptom intensity (b) and impact of CD symptoms on quality of life (c) across an injection cycle. Respondents were asked to rate the intensity of symptoms and impact of symptoms on quality of life [scale 0–10] at three different points of treatment: peak effect, waning of effect, just prior to next injection. N = 183 respondents whose symptoms reappear between two sessions of injections. BoNT-A botulinum neurotoxin type A, CD cervical dystonia
Fig. 3
Fig. 3
Re-emergent symptoms. Question: Select the [pre-existing symptoms] which reappear between two sessions of BoNT-A injections
Fig. 4
Fig. 4
Impact of symptom re-emergence on professional life (working respondents). Question: How does the reappearance of your cervical dystonia pre-existing symptoms between two sessions of BoNT-A injections affect your work? (N = 119 working respondents). BoNT-A botulinum neurotoxin type A
See this image and copyright information in PMC

References

    1. Chan J, Brin MF, Fahn S. Idiopathic cervical dystonia: clinical characteristics. Mov Disord. 1991;6(2):119–126. doi: 10.1002/mds.870060206. - DOI - PubMed
    1. Stacy M. Epidemiology, clinical presentation, and diagnosis of cervical dystonia. Neurol Clin. 2008;26(Suppl 1):23–42. doi: 10.1016/s0733-8619(08)80003-5. - DOI - PubMed
    1. Skogseid IM, Roislien J, Claussen B, Kerty E. Long-term botulinum toxin treatment increases employment rate in patients with cervical dystonia. Mov Disord. 2005;20(12):1604–1609. doi: 10.1002/mds.20670. - DOI - PubMed
    1. Ben-Shlomo Y, Camfield L, Warner T. What are the determinants of quality of life in people with cervical dystonia? J Neurol Neurosurg Psychiatry. 2002;72(5):608–614. doi: 10.1136/jnnp.72.5.608. - DOI - PMC - PubMed
    1. Pekmezovic T, Svetel M, Ivanovic N, Dragasevic N, Petrovic I, Tepavcevic DK, Kostic VS. Quality of life in patients with focal dystonia. Clin Neurol Neurosurg. 2009;111(2):161–164. doi: 10.1016/j.clineuro.2008.09.023. - DOI - PubMed

Substances