An RCT of a Patient-initiated Treatment Service for BEB and HFS

NCT02577224 | Completed DMC

• 1 other identifier: EZRD1024
• Study Type: interventional
• Target: 130
• Locations: 1 country
• Sites: 1

Brief Summary

Blepharospasm and hemifacial spasm are debilitating conditions which 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. The present study aims to compare a patient-initiated model of care, where patients determine treatment timing, with the standard model of care by using a two-arm randomised controlled trial design. It is expected that patients able to initiate their own treatment will experience less variation in disease severity and disability over a treatment cycle as they will be empowered to take the initiative when they feel they need to have treatment. A range of other outcomes will be assessed including: confidence and satisfaction in the service, depression, anxiety, quality of life and, illness and treatment beliefs. If the patient-led care model is shown to be effective in preventing distress and reducing disability in these populations; and is found to be acceptable to service users, this will have significant implications for service organisation across the NHS.

Conditions

Blepharospasm; Hemi Facial Spasm

Keywords

Patient-led care; Botulinum toxin; Dystonia; Randomised controlled trial; Acceptability

Outcome Measures

Primary Outcomes (3)

• Disease severity

  For patients with blepharospasm and hemifacial spasm, disease severity and frequency of symptoms will be assessed using the Jankovic Rating Scale (JRS), a clinician completed measure. The JRS consists of two subscales that measure severity and frequency separately. A 2-point improvement in the JRS sum score is considered a clinically relevant improvement. For patients with hemifacial spasm an additional rating scale for severity and frequency of cheek involvement will be included, which has been recommended by the questionnaire developers.

  18 months

• Disability

  The Blepharospasm Disability Index (BSDI)© is a patient self-report measure which asks patients' about six daily activities: reading, driving a vehicle, watching TV, shopping, walking and doing everyday activities. The measure is also recommended for use when assessing the impact of Botulinum toxin in hemifacial spasm. The measure has been found to possess good convergent validity with the JRS, good internal consistency (Cronbach's alpha = 0.88) and adequate test-retest reliability by its developers.

  18 months

• Patient satisfaction

  Patient satisfaction will be measured using the 8-item Client Satisfaction Questionnaire (CSQ). The scale has good internal reliability, with Cronbach's alpha ranging from 0.93 to 0.93 and good construct validity

  18 months

Secondary Outcomes (8)

• Side effects

  18 months

• Confidence in system of care

  18 months

• Patient quality of life

  18 months

• Mood

  18 months

• Illness perceptions

  18 months

Study Arms (2)

Intervention

EXPERIMENTAL

Participants randomized to the intervention group (patient-initiated treatment) will be asked to initiate their own treatment during the nine months they are taking part in the trial. Intervention group participants will receive information about when and how to initiate an appointment. Contact details for the service will be provided along with information on how quickly an appointment will be made, with whom and the procedure in the case of an emergency. All patients requesting an appointment will be booked in to the next available slot within the twice weekly ring-fenced nurse-led clinics. Any subsequent scheduled appointments will be cancelled and all future treatment will be initiated by the patient.

Other: Patient-initiated treatment

Control

NO INTERVENTION

Participants in the control group will receive treatment as usual. This consists of scheduled appointments in the hospital-based nurse-led botulinum toxin clinic. The frequency with which these appointments takes place are based on clinical judgement, but tend to range between every 6 weeks to every 4 months.

Interventions

Patient-initiated treatment OTHER

Please see Intervention Arm

Intervention

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of hemifacial spasm or blepharospasm
• Attending a hospital-based botulinum toxin clinic at Moorfields Eye Hospital NHS Foundation Trust.
• Patients stable on botulinum toxin treatment, defined as receiving toxin treatment at stable doses over two previous cycles and free from and side effects.
• Aged 18 years old and over.
• Capacity to give informed consent to participate in the study, judged by the Research Nurse.

You may not qualify if:

• Participants with significant co-morbidities (i.e. their predominant treatment is for another illness).
• Inability to communicate fluently in written and/or spoken English, to complete study measures.

Sponsors & Collaborators

• Moorfields Eye Hospital NHS Foundation Trust: lead
• Merz Pharmaceuticals: collaborator
• City, University of London: collaborator

Study Sites (1)

Moorfields Eye Hospital NHS Foundation Trust

London, EC1V 2PD, United Kingdom

Location

Related Publications (8)

• Kraft SP, Lang AE. Cranial dystonia, blepharospasm and hemifacial spasm: clinical features and treatment, including the use of botulinum toxin. CMAJ. 1988 Nov 1;139(9):837-44.

  PMID: 3052771 BACKGROUND

• Muller J, Kemmler G, Wissel J, Schneider A, Voller B, Grossmann J, Diez J, Homann N, Wenning GK, Schnider P, Poewe W; Austrian Botulinum Toxin and Dystonia Study Group. The impact of blepharospasm and cervical dystonia on health-related quality of life and depression. J Neurol. 2002 Jul;249(7):842-6. doi: 10.1007/s00415-002-0733-1.

  PMID: 12140667 BACKGROUND

• Reimer J, Gilg K, Karow A, Esser J, Franke GH. Health-related quality of life in blepharospasm or hemifacial spasm. Acta Neurol Scand. 2005 Jan;111(1):64-70. doi: 10.1111/j.1600-0404.2004.00357.x.

  PMID: 15595940 BACKGROUND

• Jinnah HA, Berardelli A, Comella C, Defazio G, Delong MR, Factor S, Galpern WR, Hallett M, Ludlow CL, Perlmutter JS, Rosen AR; Dystonia Coalition Investigators. The focal dystonias: current views and challenges for future research. Mov Disord. 2013 Jun 15;28(7):926-43. doi: 10.1002/mds.25567.

  PMID: 23893450 BACKGROUND

• Colosimo C, Tiple D, Berardelli A. Efficacy and safety of long-term botulinum toxin treatment in craniocervical dystonia: a systematic review. Neurotox Res. 2012 Nov;22(4):265-73. doi: 10.1007/s12640-012-9314-y. Epub 2012 Feb 23.

  PMID: 22359151 BACKGROUND

• 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 Serv Res. 2013 Dec 1;13:501. doi: 10.1186/1472-6963-13-501.

  PMID: 24289832 BACKGROUND

• McBain H, Shipley M, Olaleye A, Moore S, Newman S. A patient-initiated DMARD self-monitoring service for people with rheumatoid or psoriatic arthritis on methotrexate: a randomised controlled trial. Ann Rheum Dis. 2016 Jul;75(7):1343-9. doi: 10.1136/annrheumdis-2015-207768. Epub 2015 Aug 19.

  PMID: 26290587 BACKGROUND

• Lawes-Wickwar S, McBain H, Brini S, Hirani SP, Hurt CS, Flood C, Dunlop N, Solly D, Crampton B, Newman SP, Ezra DG. A patient-initiated treatment model for blepharospasm and hemifacial spasm: a randomized controlled trial. BMC Neurol. 2022 Mar 17;22(1):99. doi: 10.1186/s12883-022-02603-7.

  PMID: 35300599 DERIVED

MeSH Terms

Conditions

Blepharospasm; Dystonia

Condition Hierarchy (Ancestors)

Eyelid Diseases; Eye Diseases; Dyskinesias; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Daniel Ezra, MD FRCOph

  Moorfields Eye Hospital NHS Foundation Trust

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: HEALTH SERVICES RESEARCH
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 8, 2015
• First Posted: October 16, 2015
• Study Start: August 1, 2015
• Primary Completion: February 28, 2018
• Study Completion: February 28, 2018
• Last Updated: April 12, 2022

Record last verified: 2022-04

Locations

GB (1)