Skip to main content
Key Specialties
Cardiology Diabetology Endocrinology Gastroenterology Geriatrics and Gerontology Gynecology and Obstetrics Hematology Infectious Disease Internal Medicine Nephrology Neurology Oncology Pediatrics Respiratory Medicine Rheumatology Surgery
Congress Coverage
2024 ACC Congress 2023 ESMO Congress 2023 EASD Congress 2023 ERS Congress 2023 ESC Congress
Clinical Collections
Advances in Alzheimer’s

At a glance: The STEP trials

A round-up of the STEP phase 3 clinical trials evaluating semaglutide for weight loss in people with overweight or obesity.
ADVANCED SEARCH
Log in
Top
Trials
Published in: Trials 1/2016

Open Access 01-12-2016 | Study protocol

Effectiveness and cost-effectiveness of a patient-initiated botulinum toxin treatment model for blepharospasm and hemifacial spasm compared to standard care: study protocol for a randomised controlled trial

Authors: Sadie Wickwar, Hayley McBain, Stanton P. Newman, Shashivadan P. Hirani, Catherine Hurt, Nicola Dunlop, Chris Flood, Daniel G. Ezra

Published in: Trials | Issue 1/2016

Login to get access

Abstract

Background

Blepharospasm and hemifacial spasm are debilitating conditions that significantly impact on patient quality of life. Cyclical treatment with botulinum toxin injections offers temporary relief, but the duration of treatment efficacy is variable. The standard model of patient care defines routine fixed-time based scheduled treatment cycles which may lead to unnecessarily frequent treatment for some patients and experience of distressing symptoms in others, if symptoms return before the scheduled follow-up period.

Methods/Design

A randomised controlled trial will compare a patient-initiated model of care, where patients determine botulinum toxin treatment timing, to the standard model of care in which care is scheduled by the clinical team. A sample of 266 patients with blepharospasm or hemifacial spasm will be recruited from Moorfields Eye Hospital (MEH), London. The trial will be accompanied by a mixed-methods evaluation of acceptability of the new service. Patients who meet eligibility criteria will be assessed at baseline and those in the intervention group will be provided with instructions on how to book their own treatment appointments. Patients in both groups will be followed up 3 and 9 months into the trial and all patients will be returned to usual care after 9 months to meet safety protocols. Primary outcome measures include disease severity (questionnaire), functional disability (questionnaire) and patient satisfaction with care (questionnaire). Secondary outcomes include disease-specific quality of life (questionnaire), mood (questionnaire), illness and treatment perceptions (questionnaire and semi-structured interviews), economic impact (questionnaire) and acceptability (questionnaire and semi-structured interviews).

Discussion

This trial will assess the effectiveness and cost-effectiveness of a patient-led care model for botulinum toxin therapy. If the new model is shown to be effective in reducing distress and disability in these populations and is found to be acceptable to patients, whilst being cost-effective, this will have significant implications for service organisation across the NHS.

Trial registration

UK Clinical Research Network (UKCRN) Portfolio 18660. Clinicaltrials.gov ID NCT02577224 (registered 29 October 2015)
Literature
1.
Kraft SP, Lang AE. Cranial dystonia, blepharospasm and hemifacial spasm: clinical features and treatment, including the use of botulinum toxin. CMAJ: Canadian Medical Association Journal. 1988;139(9):837.PubMedPubMedCentral
2.
Müller J, Kemmler G, Wissel J, Schneider A, Voller B, Grossmann J, et al. The impact of blepharospasm and cervical dystonia on health-related quality of life and depression. J Neurol. 2002;249(7):842–6.CrossRefPubMed
3.
Reimer J, Gilg K, Karow A, Esser J, Franke GH. Health-related quality of life in blepharospasm or hemifacial spasm. Acta Neurologica Scandinavica. 2005;111(1):64–70.CrossRefPubMed
4.
Jinnah HA, Berardelli A, Comella C, DeFazio G, DeLong MR, Factor S, et al. The focal dystonias: current views and challenges for future research. Movement Disorders. 2013;28(7):926–43.CrossRefPubMedPubMedCentral
5.
Colosimo C, Tiple D, Berardelli A. Efficacy and safety of long-term botulinum toxin treatment in craniocervical dystonia: a systematic review. Neurotoxicity research. 2012;22(4):265–73.CrossRefPubMed
6.
Department of Health. The mandate: a mandate from the Government to the NHS Commissioning Board: April 2013 to March 2015. London: Department of Health; 2013.
7.
Department of Health. The NHS outcomes framework 2014 to 2015. London: Department of Health; 2013.
8.
Whear R, Abdul-Rahman AK, Thompson-Coon J, Boddy K, Perry MG, Stein K. Patient initiated clinics for patients with chronic or recurrent conditions managed in secondary care: a systematic review of patient reported outcomes and patient and clinician satisfaction. BMC health services research. 2013;13(1):501.CrossRefPubMedPubMedCentral
9.
McBain H, Shipley M, Moore S, Olaleye A, Hirani S, Newman SP. A patient-initiated DMARD self-monitoring service for people with rheumatoid or psoriatic arthritis on methotrexate: a randomised controlled trial. Ann Rheum Dis. 2015;1-7. doi:10.​1136/​annrheumdis-2015-207768.
10.
Oxford Radcliffe Hospital NHS Trust. Botulinum toxin follow-up clinic for treatment of blepharospasm and hemi-facial spasm. 2013. 30-10-2014.
11.
Curry LA, Krumholz HM, O'Cathain A, Clark VLP, Cherlin E, Bradley EH. Mixed methods in biomedical and health services research. Circulation: Cardiovascular Quality and Outcomes. 2013;6(1):119–23.PubMedCentral
12.
Jankovic J, Orman J. Botulinum A toxin for cranial-cervical dystonia: a double-blind, placebo-controlled study. Neurology. 1987;37(4):616.CrossRefPubMed
13.
Jankovic J, Kenney C, Grafe S, Goertelmeyer R, Comes G. Relationship between various clinical outcome assessments in patients with blepharospasm. Movement Disorders. 2009;24(3):407–13.CrossRefPubMed
14.
Wabbels B, Roggenkämper P. Botulinum toxin in hemifacial spasm: the challenge to assess the effect of treatment. Journal of neural transmission. 2012;119(8):963–80.CrossRefPubMed
15.
Goertelmeyer S, Brinkmann G, Comes A, Delcker A. The Blepharospasm Disability Index (BSDI) for the assessment of functional health in focal dystonia. Clin Neurophysiol. 2002;113 Suppl 1:S77–8.
16.
Roggenkämper P, Jost WH, Bihari K, Comes G, Grafe S. NT 201 Blepharospasm Study Team. Efficacy and safety of a new botulinum toxin type A free of complexing proteins in the treatment of blepharospasm. Journal of neural transmission. 2006;113(3):303–12.CrossRefPubMed
17.
Attkisson CC, Zwick R. The Client Satisfaction Questionnaire: psychometric properties and correlations with service utilization and psychotherapy outcome. Evaluation and program planning. 1982;5(3):233–7.CrossRefPubMed
18.
Clifford C, Greenfield T. The UCSF Client Satisfaction Scales: I. The Client Satisfaction Questionnaire-8. In: Mahwah NJ, editor. The use of psychological testing for treatment planning and outcomes assessment, vol. 3. 3rd ed. New Jersey, USA: Lawrence Erlbaum Associates; 2004.
19.
Müller J, Wissel J, Kemmler G, Voller B, Bodner T, Schneider A, et al. Craniocervical dystonia questionnaire (CDQ-24): development and validation of a disease-specific quality of life instrument. Journal of Neurology, Neurosurgery & Psychiatry. 2004;75(5):749–53.CrossRef
20.
Malhotra R, Then SY, Richards A, Cheek E. The effect of Bangerter occlusion foils on blepharospasm and hemifacial spasm in occlusion-positive and occlusion-negative patients. The open ophthalmology journal. 2010;4:1.CrossRefPubMedPubMedCentral
21.
Hirani SP, Patterson DL, Newman SP. What do coronary artery disease patients think about their treatments? An assessment of patients’ treatment representations. Journal of health psychology. 2008;13(3):311–22.CrossRefPubMed
22.
Cosco TD, Doyle F, Ward M, McGee H. Latent structure of the Hospital Anxiety and Depression Scale: a 10-year systematic review. Journal of Psychosomatic Research. 2012;72:180–4.CrossRefPubMed
23.
Zigmond AS, Snaith RP. The hospital anxiety and depression scale. Acta psychiatrica scandinavica. 1983;67(6):361–70.CrossRefPubMed
24.
Kirwan JR, Mitchell K, Hewlett S, Hehir M, Pollock J, Memel D, et al. Clinical and psychological outcome from a randomized controlled trial of patient-initiated direct-access hospital follow-up for rheumatoid arthritis extended to 4 years. Rheumatology. 2003;42(3):422–6.CrossRefPubMed
25.
Moss-Morris R, Weinman J, Petrie K, Horne R, Cameron L, Buick D. The revised illness perception questionnaire (IPQ-R). Psychology and Health. 2002;17(1):1–16.CrossRef
26.
Hewlett S, Mitchell K, Haynes J, Paine T, Korendowych E, Kirwan JR. Patient-initiated hospital follow-up for rheumatoid arthritis. Rheumatology. 2000;39(9):990–7.CrossRefPubMed
27.
Hewlett S, Kirwan J, Pollock J, Mitchell K, Hehir M, Blair PS, et al. Patient initiated outpatient follow up in rheumatoid arthritis: six year randomised controlled trial. BMJ. 2005;330(7484):171.CrossRefPubMedPubMedCentral
28.
Sekhon M, Cartwright M, Francis JJ. How has acceptability of healthcare interventions been defined and assessed? An overview of systematic reviews. Nottingham: 10th UK Society for Behavioural Medicine Annual Scientific Meeting; 4 December 2014.
29.
Beecham JK, Knapp MRJ. Costing psychiatric interventions. In: Thornicroft G, Brewin C, Wing JK, editors. Measuring Mental Health Needs. London: Gaskell; 1992.
30.
Chisholm D, Knapp M, Knudsen HC, et al. Client socio-demographic and service receipt inventory – European version: development of an instrument for international research. British Journal of Psychiatry. 2000;177(39):S28–33.CrossRef
31.
Byford S, Leese M, Knapp M, Seivewright H, Cameron S, Jones V, et al. Comparison of alternative methods of collection of service use data for the economic evaluation of health care interventions. Health economics. 2007;16(5):531–6.CrossRefPubMed
32.
Borenstein M. Power and Precision™ (Version 4.1) [Software]. USA: Biostat, Inc; 2011.
33.
Maas CJ, Hox JJ. Sufficient sample sizes for multilevel modeling. Methodology: European Journal of Research Methods for the Behavioral and Social Sciences. 2005;1(3):86.CrossRef
34.
Francis JJ, Johnston M, Robertson C, Glidewell L, Entwistle V, Eccles MP, et al. What is an adequate sample size? Operationalising data saturation for theory-based interview studies. Psychology and Health. 2010;25(10):1229–45.CrossRefPubMed
35.
Heck R. Multilevel and longitudinal modeling with IBM SPSS. New York: Routledge; 2014.
36.
Turner HM, Bernard RM. Calculating and synthesizing effect sizes. Contemporary Issues in Communication Science and Disorders. 2006;33:42–55.
37.
Cohen J. Statistical power analysis for the behavioral sciences. 2nd ed. New Jersey: Lawrence Erlbaum Associates; 1988.
38.
Ritchie J, Spencer L. Qualitative data analysis for applied policy research. In: Huberman AM, Miles MB, editors. The qualitative researcher’s companion. California: Sage; 2002.
39.
International QSR. NVivo (Version 10) [Software]. 2014.
Metadata
Title
Effectiveness and cost-effectiveness of a patient-initiated botulinum toxin treatment model for blepharospasm and hemifacial spasm compared to standard care: study protocol for a randomised controlled trial
Authors
Sadie Wickwar
Hayley McBain
Stanton P. Newman
Shashivadan P. Hirani
Catherine Hurt
Nicola Dunlop
Chris Flood
Daniel G. Ezra
Publication date
01-12-2016
Publisher
BioMed Central
Published in
Trials / Issue 1/2016
Electronic ISSN: 1745-6215
DOI
https://doi.org/10.1186/s13063-016-1263-y

Other articles of this Issue 1/2016

Trials 1/2016 Go to the issue

Research

Exploring methods the for selection and integration of stakeholder views in the development of core outcome sets: a case study in reconstructive breast surgery

Study protocol

Efficacy of transcranial direct-current stimulation (tDCS) in women with provoked vestibulodynia: study protocol for a randomized controlled trial

Study protocol

Investigating the feasibility and acceptability of a cognitive behavioural suicide prevention therapy for people in acute psychiatric wards (the ‘INSITE’ trial): study protocol for a randomised controlled trial

Commentary

Ethnographic methods for process evaluations of complex health behaviour interventions

Study protocol

The effectiveness of the paclitaxel-coated Luminor® balloon catheter versus an uncoated balloon catheter in superficial femoral and popliteal arteries in preventing vessel restenosis or reocclusion: study protocol for a randomized controlled trial

Study protocol

Prevention of catheter-related bladder discomfort – pudendal nerve block with ropivacaine versus intravenous tramadol: study protocol for a randomized controlled trial