Efficacy of Botulinum Toxin for the Treatment of Dystonia Associated With Parkinson's Disease and Atypical Parkinsonism

Efficacy of Botulinum Toxin for the Treatment of Dystonia Associated With Parkinson's Disease and Atypical Parkinsonism: a Monocentric Cohort Study With Patient Reported Outcome

Dystonia is a disabling symptom affecting both patients with idiopathic Parkinson's disease (PD) and atypical parkinsonism (AP).

Botulinum toxinum (BoNT), by blocking muscle contraction, is a possible treatment for focal dystonia.

The benefit of BoNT treatment has been proven in some focal dystonia associated with PD or AP.

The investigators aim to give an overview of the efficacy of BoNT in a variety of focal dystonia in a large cohort of parkinsonian patients.

Study Overview

• Status: Completed
• Conditions: Parkinson Disease; Dystonia; Atypical Parkinsonism

• Study Type: Observational
• Enrollment (Actual): 63

Contacts and Locations

Study Locations

• France
  • Seine-Saint-Denis
    • Bobigny, Seine-Saint-Denis, France, 93009
      • Avicenne Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

All patients that have been evaluated in the Neurology department of Avicenne Hospital (academic medical center) between september 1st 2017 and february 11th 2021.

Data will be retrospectively collected within the medical records of those patients.

Description

Inclusion Criteria:

• Diagnosed Parkinson's disease or Atypical Parkinsonism
• Age > 18 years
• Reported dystonia
• At least 1 injection with Botulinum toxin realized

Exclusion Criteria:

• Refusal to participate in the study

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
Patients with dystonia secondary to atypical Parkinsonism or Parkinson's disease; Patients with dystonia secondary to idiopathic Parkinson's disease or atypical parkinsonism and treated with BoNT
Patients with atypical Parkinsonism or Parkinson's disease and no dystonia; Patients with idiopathic Parkinson's disease or atypical parkinsonism and without reported dystonia

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Comparison of the patient reported efficacy of botulinum toxin treatment between PD and AP patients	Evaluation by the patient of the efficacy of the former injection on an improvement scale ranging from 0 to 100% (minimum value : 0, maximum value : 100, higher scores mean a better outcome) systematically made in routine care during follow up appointment	Up to twelve months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Comparison of the patient reported duration of improvement after treatment by botulinum toxin between PD and AP patients	Evaluation by the patient of the duration of the period during which symptoms were improved (in weeks) systematically made in routine care during follow up appointment	Up to twelve months

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Comparison of the patients' age between PD and AP groups	Age in years of patients from PD and AP groups	Baseline
Comparison of the sex-ratio between PD and AP groups	Percentage of female (%) in PD and AP groups	Screening
Comparison of the patients' duration of disease between PD and AP groups	Duration of the disease (years since diagnosis of PD or AP) of patients from PD and AP groups	Baseline
Comparison of the patients' severity of the disease between PD and AP groups	Severity of the disease in both PD and AP groups measured by the Hoehn and Yahr scale (stage 0 to stage 5, a higher score meaning a more severe disease)	Baseline
Comparison of the patients' daily need in levodopa between PD and AP groups	Levodopa equivalent daily dose (in mg, method as described by CL Tomlinson) of patient from PD and AP groups	Baseline
Comparison of the percentage of patients treated with an apomorphine pump between PD and AP groups	Percentage of patients treated with apomorphine (%) in PD and AP groups	Baseline
Comparison of the percentage of patients treated with deep brain stimulation between PD and AP groups	Percentage of patients treated with deep brain stimulation (%) in PD and AP groups	Baseline
Comparison of the patients' age between patients presenting with dystonia and patients without reported dystonia	Age in years	Baseline
Comparison of the percentage of patients treated with deep brain stimulation between patients presenting with dystonia and patients without reported dystonia	Percentage of patients treated with deep brain stimulation (%) in both groups	Baseline
Comparison of the percentage of patients treated with an apomorphine pump between patients presenting with dystonia and patients without reported dystonia	Percentage of patients treated with apomorphine (%) in both groups	Baseline
Comparison of the patients' daily need in levodopa between patients presenting with dystonia and patients without reported dystonia	Levodopa equivalent daily dose (in mg, method as described by CL Tomlinson) of patient from both groups	Baseline
Comparison of the severity of the disease between patients presenting with dystonia and patients without reported dystonia	Severity of the disease in both groups measured by the Hoehn and Yahr scale (stage 0 to stage 5, a higher score meaning a more severe disease)	Baseline
Comparison of the patients' duration of disease between patients presenting with dystonia and patients without reported dystonia	Duration of the disease (years since diagnosis of AP or PD) of patients	Baseline
Comparison of the sex-ratio between patients presenting with dystonia and patients without reported dystonia	Percentage of female (%)	Baseline
Comparison of the dystonia's characteristics between PD and AP groups : muscle affected	Muscle affected by dystonia (described with International Anatomical Terminology)	Day 0
Comparison of the dystonia's characteristics between PD and AP groups : duration of dystonia	Duration of the dystonia (years since first report of the dystonia)	Day 0
Comparison of the characteristics of the botulinum toxin injection between PD and AP groups : dose	Dose of BoNT used (in mL)	Day 0
Comparison of the characteristics of the botulinum toxin injection between PD and AP groups : type of BoNT	Type of botulinum toxin used (onabotulinumtoxinA, abobotulinumtoxinA or incobotulinumtoxinA)	Day 0
Comparison of the characteristics of the botulinum toxin injection between PD and AP groups : site of injection	Site of injection (described with International Anatomical Terminology)	Day 0
Comparison of the characteristics of the botulinum toxin injection between PD and AP groups : number of injections	Number of injection performed during injection appointment	Day 0
Comparison of the characteristics of the botulinum toxin injection between PD and AP groups : usage of electromyographic or ultrasound guidance	Usage of ultrasound or electromyography to guide BoNT injection	Day 0

Collaborators and Investigators

• Sponsor: Hospital Avicenne
• Collaborators: Assistance Publique - Hôpitaux de Paris
• Investigators: Principal Investigator: Bertrand Degos, MD,PhD, Hospital AVICENNE

Publications and helpful links

General Publications

• Dressler D. Botulinum toxin drugs: brief history and outlook. J Neural Transm (Vienna). 2016 Mar;123(3):277-9. doi: 10.1007/s00702-015-1478-1. Epub 2015 Nov 11.
• Jankovic J. Botulinum toxin: State of the art. Mov Disord. 2017 Aug;32(8):1131-1138. doi: 10.1002/mds.27072. Epub 2017 Jun 22.
• Tolosa E, Compta Y. Dystonia in Parkinson's disease. J Neurol. 2006 Dec;253 Suppl 7:VII7-13. doi: 10.1007/s00415-006-7003-6.
• Marsili L, Bologna M, Kojovic M, Berardelli A, Espay AJ, Colosimo C. Dystonia in atypical parkinsonian disorders. Parkinsonism Relat Disord. 2019 Sep;66:25-33. doi: 10.1016/j.parkreldis.2019.07.030. Epub 2019 Jul 23.
• Mills R, Bahroo L, Pagan F. An update on the use of botulinum toxin therapy in Parkinson's disease. Curr Neurol Neurosci Rep. 2015 Jan;15(1):511. doi: 10.1007/s11910-014-0511-3.
• Pacchetti C, Albani G, Martignoni E, Godi L, Alfonsi E, Nappi G. "Off" painful dystonia in Parkinson's disease treated with botulinum toxin. Mov Disord. 1995 May;10(3):333-6. doi: 10.1002/mds.870100317.
• Giladi N, Meer J, Honigman S. The use of botulinum toxin to treat "striatal" toes. J Neurol Neurosurg Psychiatry. 1994 May;57(5):659. doi: 10.1136/jnnp.57.5.659. No abstract available.
• Rieu I, Degos B, Castelnovo G, Vial C, Durand E, Pereira B, Simonetta-Moreau M, Sangla S, Fluchere F, Guehl D, Burbaud P, Geny C, Gayraud D, Ory-Magne F, Bouhour F, Llinares E, Derost P, Marques A, Durif F. Incobotulinum toxin A in Parkinson's disease with foot dystonia: A double blind randomized trial. Parkinsonism Relat Disord. 2018 Jan;46:9-15. doi: 10.1016/j.parkreldis.2017.10.009. Epub 2017 Oct 19.
• Lapostolle A, Houot M, Mongin M, Degos B. Comparison of Botulinum neurotoxin efficiency in dystonia associated with Parkinson's disease and atypical parkinsonism: a retrospective study with a self-reported improvement scale. J Neurol. 2022 Nov;269(11):6021-6028. doi: 10.1007/s00415-022-11280-y. Epub 2022 Jul 19.

Study record dates

Study Major Dates

• Study Start (Actual): September 1, 2017
• Primary Completion (Actual): February 11, 2021
• Study Completion (Actual): February 11, 2021

Study Registration Dates

• First Submitted: June 6, 2021
• First Submitted That Met QC Criteria: June 24, 2021
• First Posted (Actual): July 2, 2021

Study Record Updates

• Last Update Posted (Actual): July 2, 2021
• Last Update Submitted That Met QC Criteria: June 24, 2021
• Last Verified: June 1, 2021

More Information

Terms related to this study

• Keywords: Parkinson Disease; Dystonia; Atypical Parkinsonism; Botulinum toxinum
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Basal Ganglia Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases; Dyskinesias; Parkinson Disease; Dystonia; Dystonic Disorders; Parkinsonian Disorders
• Other Study ID Numbers: CLEA-2021-167

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No