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

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Brief Title

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

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.
    


Study Type

Interventional


Primary Outcome

Disease severity

Secondary Outcome

 Side effects

Condition

Blepharospasm

Intervention

Patient-initiated treatment

Study Arms / Comparison Groups

 Intervention
Description:  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.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Other

Estimated Enrollment

130

Start Date

August 2015

Completion Date

October 2017

Primary Completion Date

October 2017

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.
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Daniel Ezra, MD FRCOph, , 

Location Countries

United Kingdom

Location Countries

United Kingdom

Administrative Informations


NCT ID

NCT02577224

Organization ID

EZRD1024


Responsible Party

Sponsor

Study Sponsor

Moorfields Eye Hospital NHS Foundation Trust

Collaborators

 Merz Pharmaceuticals

Study Sponsor

Daniel Ezra, MD FRCOph, Principal Investigator, Moorfields Eye Hospital NHS Foundation Trust


Verification Date

September 2016