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

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Randomised Controlled Trial to Explore the Effectiveness and Cost-effectiveness of a Patient-initiated Botulinum Toxin Treatment Model for Blepharospasm and Hemifacial Spasm

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.

Detailed Summary:

Blepharospasm is a dystonia described by sustained, forced, involuntary closure of both eyelids, caused by muscle contraction. Hemifacial spasm occurs on one side of the face and can result in complete closure of one eye, and spasms across the cheek, face and neck. Hemifacial spasm, as its name implies, is unilateral, whereas blepharospasm always affects both eyes. Blepharospasm and hemifacial spasm are debilitating conditions, which carry the risk of functional blindness and can lead to appearance concerns, social embarrassment and isolation, depression and poor quality of life.[1-3]

Botulinum toxin is the treatment used in standard care to stop spasms, but results in a fluctuating pattern of relief and aggravation[4] and hence patients return for repeated injections. A recent systematic review indicated that the patient reported benefits of botulinum toxin in blepharospasm ranged from no improvement to 96% of patients reporting a significant relief in symptoms.[5] Evidence for the duration of benefit provided by repeated treatment is was also markedly inconsistant.[5] Although the definition of benefit did differ between studies in this review, research cannot explain such inconsistencies. Despite this a standardised treatment regimen, of injections on average 3 times a year, is currently used across all patients at Moorfields Eye Hospital and is typical throughout the UK. This may mean that some people are left experiencing debilitating symptoms until their next scheduled appointment and some are being seen too often, hence alternative models of care need to be considered.

Patient-centred care is at the forefront of the NHS,[6;7] reflecting the shift away from the paternalistic model of healthcare. Patients are now encouraged to take a more active role in knowing and managing their health, and this is especially importa
Sponsor: Moorfields Eye Hospital NHS Foundation Trust

Current Primary Outcome:

• Disease severity [ Time Frame: 18 months ]
  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.
• Disability [ Time Frame: 18 months ]
  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.
• Patient satisfaction [ Time Frame: 18 months ]
  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

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Side effects [ Time Frame: 18 months ]
  Individual side effects including ptosis, tearing, blurred vision, double vision, hematoma, foreign body sensation, will be recorded at each clinic visit. The total number of side effects will be recorded for each patient.
• Confidence in system of care [ Time Frame: 18 months ]
  Confidence in the system of care will be assessed using a 10cm visual analogue scale (VAS). Patients will be asked 'How confident are you that if you required treatment this system of care would be able to support you?' ranging from 'not at all confident' to 'completely confident'.
• Quality of life [ Time Frame: 18 months ]
  Quality of life will be measured using the Craniocervical dystonia questionnaire (CDQ-24) which was developed and validated in patients with blepharospasm, and has also been used with success in patients with hemifacial spasm. This 24 item measure assesses quality of life across five domains: stigma, emotional well-being, pain, activities of daily living and social/family life. The measure has been found to possess good internal consistency with Cronbach's alphas ranging from 0.77 to 0.89, good construct validity when compared who the SF-36, good discriminant validity and test retest reliability.
• Mood [ Time Frame: 18 months ]
  The Hospital Anxiety and Depression Scale (HADS) will be used to assess mood. The HADS is a 14-item self-screening questionnaire for depression and anxiety in patients with physical health problems. The two 7-item subscales measure how a person has been feeling in the past week. The scale provides cut-off scores for the presence of potentially clinical levels of anxiety and depression. A systematic review of the HADS has confirmed the factor structure, found the cut-off points to be valid against clinical interviews, reports excellent internal consistency (Cronbach's alpha: anxiety 0.68-0.93; depression 0.67-0.90)
• Illness perceptions [ Time Frame: 18 months ]
  Illness perceptions are cognitive representations or beliefs that a patient has about their illness. These concepts will be measured using the revised illness perceptions questionnaire (IPQ-R), which has been widely used and is a valid and reliable measure.
• Treatment beliefs [ Time Frame: 18 months ]
  Beliefs about botulinum toxin will be measured using the Treatment Representations Inventory (TRI). A 27-item measure, consisting of four subscales including 'treatment-value', 'treatment-concerns', 'decision-satisfaction' and 'cure'.
• Acceptability [ Time Frame: 18 months ]
  Acceptability of the new patient-initiated service and standard care will be measured using the 7-item Acceptability Questionnaire developed by the study team.
• Cost effectiveness [ Time Frame: 18 months ]
  An adapted version of the Client Service Receipt Inventory (CSRI) has been employed, which has been widely employed in previous studies, particularly in the mental health setting. The impact of the service on direct and indirect costs will be estimated at the end of the trial period.

Original Secondary Outcome:

• Side effects [ Time Frame: 18 months ]
  Individual side effects including ptosis, tearing, blurred vision, double vision, hematoma, foreign body sensation, will be recorded at each clinic visit. The total number of side effects will be recorded for each patient.
• Confidence in system of care [ Time Frame: 18 months ]
  Confidence in the system of care will be assessed using a 10cm visual analogue scale (VAS). Patients will be asked 'How confident are you that if you required treatment this system of care would be able to support you?' ranging from 'not at all confident' to 'completely confident'.
• Quality of life [ Time Frame: 18 months ]
  Quality of life will be measured using the Craniocervical dystonia questionnaire (CDQ-24) which was developed and validated in patients with blepharospasm, and has also been used with success in patients with hemifacial spasm. This 24 item measure assesses quality of life across five domains: stigma, emotional well-being, pain, activities of daily living and social/family life. The measure has been found to possess good internal consistency with Cronbach's alphas ranging from 0.77 to 0.89, good construct validity when compared who the SF-36, good discriminant validity and test retest reliability.
• Mood [ Time Frame: 18 months ]
  The Hospital Anxiety and Depression Scale (HADS) will be used to assess mood. The HADS is a 14-item self-screening questionnaire for depression and anxiety in patients with physical health problems. The two 7-item subscales measure how a person has been feeling in the past week. The scale provides cut-off scores for the presence of potentially clinical levels of anxiety and depression. A systematic review of the HADS has confirmed the factor structure, found the cut-off points to be valid against clinical interviews, reports excellent internal consistency (Cronbach's alpha: anxiety 0.68-0.93; depression 0.67-0.90)
• Illness perceptions [ Time Frame: 18 months ]
  Illness perceptions are cognitive representations or beliefs that a patient has about their illness. These concepts will be measured using the revised illness perceptions questionnaire (IPQ-R), which has been widely used and is a valid and reliable measure.
• Treatment beliefs [ Time Frame: 18 months ]
  Beliefs about botulinum toxin will be measured using the Treatment Representations Inventory (TRI). A 27-item measure, consisting of four subscales including 'treatment-value', 'treatment-concerns', 'decision-satisfaction' and 'cure'.
• Acceptability [ Time Frame: 18 months ]
  Acceptability of the new patient-initiated service and standard care will be measured using the 7-item Acceptability Questionnaire developed by the study team.
• Economic impact [ Time Frame: 18 months ]
  An adapted version of the Client Service Receipt Inventory (CSRI) has been employed, which has been widely employed in previous studies, particularly in the mental health setting. The impact of the service on direct and indirect costs will be estimated at the end of the trial period.

Dates:
Date Received: May 8, 2015
Date Started: August 2015
Date Completion: October 2017
Last Updated: March 21, 2017
Last Verified: September 2016