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.
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
  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).
  
  
  CONCLUSIONS
  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.
  
  
  BACKGROUND
  UK Clinical Research Network (UKCRN) Portfolio 18660. Clinicaltrials.gov ID NCT102577224 (registered 29 October 2015).
authors.affiliation: Centre for Health Services Research, School of Health Sciences, City University London, London, UK. sadie.wickwar.1@city.ac.uk.|Moorfields Eye Hospital, London, UK. sadie.wickwar.1@city.ac.uk.
authors.forename: Sadie
authors.initials: S
authors.lastname: Wickwar
authors.affiliation: Centre for Health Services Research, School of Health Sciences, City University London, London, UK. hayley.mcbain.1@city.ac.uk.|Community Health Newham, East London Foundation Trust, London, UK. hayley.mcbain.1@city.ac.uk.
authors.forename: Hayley
authors.initials: H
authors.lastname: McBain
authors.affiliation: Centre for Health Services Research, School of Health Sciences, City University London, London, UK. stanton.newman.1@city.ac.uk.
authors.forename: Stanton P
authors.initials: SP
authors.lastname: Newman
authors.affiliation: Centre for Health Services Research, School of Health Sciences, City University London, London, UK. shashi.hirani.1@city.ac.uk.
authors.forename: Shashivadan P
authors.initials: SP
authors.lastname: Hirani
authors.affiliation: Centre for Health Services Research, School of Health Sciences, City University London, London, UK. catherine.hurt.1@city.ac.uk.
authors.forename: Catherine
authors.initials: C
authors.lastname: Hurt
authors.affiliation: Moorfields Eye Hospital, London, UK. nicola.dunlop@moorfields.nhs.uk.
authors.forename: Nicola
authors.initials: N
authors.lastname: Dunlop
authors.affiliation: Centre for Health Services Research, School of Health Sciences, City University London, London, UK. c.flood@city.ac.uk.
authors.forename: Chris
authors.initials: C
authors.lastname: Flood
authors.affiliation: Moorfields Eye Hospital, London, UK. d.ezra@ucl.ac.uk.
authors.forename: Daniel G
authors.initials: DG
authors.lastname: Ezra
mesh_terms: D065087:Acetylcholine Release Inhibitors / Q000008:administration & dosage* / Q000009:adverse effects / Q000191:economics*; D000339:Affect; D001071:Appointments and Schedules; D001764:Blepharospasm / Q000175:diagnosis / Q000188:drug therapy* / Q000191:economics*; D001905:Botulinum Toxins / Q000008:administration & dosage* / Q000009:adverse effects / Q000191:economics*; D002985:Clinical Protocols; D017281:Cost of Illness; D003362:Cost-Benefit Analysis; D004185:Disability Evaluation; D004334:Drug Administration Schedule; D016527:Drug Costs; D019569:Hemifacial Spasm / Q000175:diagnosis / Q000188:drug therapy* / Q000191:economics*; D006801:Humans; D007407:Interviews as Topic; D008131:London; D010358:Patient Participation; D017060:Patient Satisfaction; D011788:Quality of Life; D012107:Research Design; D012720:Severity of Illness Index; D011795:Surveys and Questionnaires; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D065087:Acetylcholine Release Inhibitors; D001905:Botulinum Toxins