Clinical trial NCT02577224: An RCT of a Patient-initiated Treatment Service for BEB and HFS
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 Compared to Treatment as Usual
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Blepharospasm
Hemi Facial Spasm
Keywords: Patient-led care
Botulinum toxin
Dystonia
Randomised controlled trial
Acceptability
Lead sponsor: Moorfields Eye Hospital NHS Foundation Trust
Sponsor class: OTHER
Collaborators: name: Merz Pharmaceuticals
name: City, University of London
First posted: 2015-10-16
Enrollment: 130
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: HEALTH_SERVICES_RESEARCH

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 description: 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 important in conditions such as dystonia where the reality of living with the condition is demanding. As a result patient-led healthcare services are becoming increasingly more common and have the potential to address the inconsistencies found in the current botulinum toxin treatment regimen.

Research studies trialling patient-initiated services, where the patient rather than healthcare professional initiates treatment and care, have shown promise. These services provide patients with information on when and how to access services, rather than having regular scheduled appointments. A systematic review conducted by Whear et al\[8\] synthesised the evidence for this model of care across three conditions: irritable bowel disease, breast cancer and rheumatoid arthritis. Overall, there were few differences in psychological or health-related quality of life between those initiating their own outpatient follow-up appointments compared to standard care, despite in many cases patients having less contact with healthcare professionals. Patient and clinician satisfaction were also significantly greater in the patient-initiated services compared to regular appointment scheduling. Since this systematic review members of the research team have led on the development and evaluation of a patient-initiated follow-up service for patients with arthritis. Using a mixed methods study design the service was found to reduce healthcare utilisation in a RCT, without compromising clinical or psychosocial well-being, and was found to be acceptable to patients in the embedded qualitative study.\[9\]

Adopting a patient-led model of care could reduce discomfort and disability in patients with a short-term response to botulinum toxin and reduce unnecessary hospital visits and treatment for patients with a longer-term response. There is currently one patient-initiated, nurse-led botulinum toxin clinic running in the UK for patients with blepharospasm or hemifacial spasm,\[10\] but is yet to be evaluated in comparison to usual care. Due to the variable nature of treatment response blepharospasm and hemifacial spasm are appropriate conditions in which to evaluate a service of this nature. This study therefore, provides a unique opportunity to empower patients with dystonia to take control of their treatment and optimise the effects of botulinum toxin, by allowing them to seek treatment when they feel it is necessary rather than it being dictated by the clinical team, within the context of a methodologically robust evaluative.

Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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.

Primary outcomes:
measure: Disease severity
timeFrame: 18 months
measure: Disability
timeFrame: 18 months
measure: Patient satisfaction
timeFrame: 18 months
Secondary outcomes:
measure: Side effects
timeFrame: 18 months
measure: Confidence in system of care
timeFrame: 18 months
measure: Patient quality of life
timeFrame: 18 months
measure: Mood
timeFrame: 18 months
measure: Illness perceptions
timeFrame: 18 months
measure: Treatment beliefs
timeFrame: 18 months
measure: Acceptability
timeFrame: 18 months
measure: Cost effectiveness
timeFrame: 18 months
Other outcomes:
Locations:
city: London
country: United Kingdom
facility: Moorfields Eye Hospital NHS Foundation Trust
Study officials:
affiliation: Moorfields Eye Hospital NHS Foundation Trust
name: Daniel Ezra, MD FRCOph
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.