Selective Dorsal Rhizotomy (SDR) Database
Condition: Cerebral Palsy
MeSH terms: Cerebral Palsy
Study type: Observational [Patient Registry]

Summary: 
      A national database is being set-up to collate clinical outcome information before and after
      children undergo Selective Dorsal Rhizotomy (SDR). SDR is a surgical procedure that has been
      used for many years but has not been routinely available within the United Kingdom National
      Health Service (NHS). The SDR procedure is used to improve spasticity in children with
      cerebral palsy and involves dividing some of the nerve fibres running from the muscles back
      to the spinal cord. While it is believed that SDR is effective, there are no definitive data.
      In July 2014 NHS England commissioned five NHS Trusts to undertake SDR surgery in eligible
      children for a two-year period, and to collect detailed information on their outcomes. The
      proposed database will aggregate these data from the five centres and analyse them to provide
      overall evidence on the clinical effectiveness of SDR. This information will be used to
      inform future commissioning decisions. The database has been developed by the King's
      Technology Evaluation Centre (KiTEC) in conjunction with NHS England and the national
      clinical SDR steering committee and its data subcommittee. KiTEC undertakes research
      evaluations for the National Institute for Health and Care Excellence (NICE), and has been
      commissioned by NICE to undertake this work. Clinicians in the SDR data subgroup have liaised
      closely with KiTEC to agree which data to collect so that it the least amount needed and will
      be in a standardised format across all centres. When all two-year outcome data are recorded,
      KiTEC will analyse the data and present a report to NHS England, NICE and the SDR steering
      committee and data sub-group. While there are no firm plans to follow the children beyond two
      years post-surgery, all parties involved are hoping that further funding can be obtained to
      continue following these children up as these data will be unique.
    

Detailed description: 
      Disease overview:

      Cerebral Palsy is an all-encompassing term for numerous neurological conditions caused by
      problems in the brain and nervous system. More specifically, cerebral palsy is related to
      problems in muscle movement, which result in abnormalities in walking, posture, and balance.
      Other important functions can also be affected, such as language/communication, learning, and
      vision. There is currently no cure for cerebral palsy; however, there are several treatment
      options that can aid alleviation of symptoms and improve quality of life. The selection of
      appropriate treatment(s) for each child requires input from a multi-disciplinary team of
      health professionals, such as paediatricians, social workers, physiotherapists, speech and
      language therapists, occupational therapists, neurologists and surgeons.

      Treatment overview:

      The National Institute for Health and Care Excellence (NICE) published guidance related to
      treatment options for cerebral palsy in 2012 (CG145). One of the treatment options discussed
      was Selective Dorsal Rhizotomy (SDR). SDR is an irreversible surgical procedure, which
      involves the division of some of the sensory nerves of the spinal cord in order to reduce
      sensory input to the sensory-motor reflex arcs. Intensive physiotherapy is required SDR post
      surgery for up to 24 months to achieve the outcome goals of improvement in muscle function,
      tone, and quality of life. NICE CG145 recommends consideration of SDR in children with a
      GMFCS level II or III. This recommendation was based on a review of evidence evaluation,
      which concluded that there was reasonable evidence that SDR combined with appropriate
      post-operative treatment can result in significant improvements in motor function and quality
      of life.

      Study Overview:

      This current project is being commissioned by NICE to support NHS England in its
      Commissioning through Evaluation (CTE) approach. CTE was launched in September 2013 and
      involves funding medical treatments and technologies not routinely commissioned within the
      NHS. SDR is one such procedure which was selected for CTE, with the aim of evaluating the
      'outcome of SDR and demonstrate improvement in function after SDR at 6 months that is
      maintained or improved at 12 months and two years post operation'. As part of this programme,
      NICE commissioned, in early 2014, a data collection and register development service from one
      of its External Assessment Centres (EACs): King's Imaging Technology Evaluation Centre
      (KiTEC). The key objective of the KiTEC EAC is to collect SDR related data that will inform
      clinical effectiveness and other outcomes of interest to NHS England in making its
      commissioning plans and addressing the following research question from section 4.5 of CG145:

      'Does selective dorsal rhizotomy followed by intensive rehabilitation performed between the
      ages of 3 and 9 years in children who are at GMFCS level II or III result in good community
      mobility as a young adult?'

      Study Design:

      This is a multi-centre national database (register) project to obtain the evidence related to
      SDR that will address the research question in section 4.5 of CG145 and inform clinical
      effectiveness and other outcomes of interest to NHS England. All patients will undergo
      pre-assessment prior to undergoing SDR. This varies between centres, and can be anywhere from
      6 months prior to SDR. Hospital admission for SDR usually is between 1-2 weeks. Post-SDR
      follow-up will be conducted up to two years for the purposes of this project. All patients'
      will followed up to two years post-SDR by members of the relevant clinical team. Post-SDR
      discharge there will be three main data collection points for the purposes of this project:
      6, 12 and 24 months post-SDR. Patient data will be collected at various time-points, as
      agreed between the EAC and various SDR subgroup members. Pre-assessments may be split into
      more than one appointment, depending on the particular patient care pathway at each
      participating centre.

      Parents of patients will be approached by the SDR clinical team when they attend for their
      first pre-SDR clinic visit. It will be explained to the parents and children that their
      hospital will be making clinical assessments before and after SDR to enable them to provide
      optimum care and to evaluate the success of the operation and post-operative rehabilitation.
      These assessments along with patient-reported outcomes, such as quality of life assessments,
      will be standardised across all five commissioned hospitals. The parents and children will be
      asked for permission to add their data to the national database to facilitate aggregation of
      results across the United Kingdom (UK).

      Study information sheets will be provided. If they agree to take part, parents will sign a
      consent form at the 2nd pre-SDR visit. This will allow time for them to consider whether or
      not they wish to take part.

      Centre and Subject Compliance:

      Centre compliance refers to all centres providing the agreed standardised dataset and input
      into the standardised database as per the CTE selection criteria. The EAC will regularly
      monitor Centre compliance in liaison with NHS England and include tables of compliance in the
      project report. The project report (with potential for interim analysis) will include tables
      on the number of eligible patients that were referred for treatment through the
      multi-disciplinary team process and consented to part of the database as well as those that
      were actually registered in the SDR database. The project report (with potential for interim
      analysis) will list the percentage of missing data in each of the clinical domains and
      differentiate between baseline and follow-up variables. Depending on the stage of the
      database a table depicting the number of children that have been lost to follow up will be
      included in the project report or in potential interim analyses reports. The SDR centres will
      be given regular reports on the quality of their data in terms of missing variables and
      possible outliers.

      Statistical Considerations:

      The EAC statisticians will carry out all analyses. All data obtained will be analysed at
      regular specified time points to inform ongoing assessment of the clinical effectiveness,
      relevant outcomes and assessment of the overall research question.The SDR database is
      expected to have small numbers, in the region of up to n=120 a year in England. A range of
      clinical and patient-reported outcomes will be recorded and there will be no single primary
      or secondary outcome. Rather the aim is to describe and quantify the changes in a number of
      important clinical domains such as spasticity, function, range of motion, gait and quality of
      life.

      Ethical Considerations:

      As the database constitutes research involving human subjects, the following ethical
      considerations arise:

        -  Parental consent will be required to add the child's data to the national database and
           patient information sheets (PIS) have been developed for both children and parents of
           potential children at the designated SDR centres.

        -  Only the minimum number of data items needed to meet the objectives of the database and
           comply with ethical and regulatory requirements will be included in the final dataset.

        -  Children's confidentiality will be protected during data collection and handling thus
           reducing the risk of disclosure.
    

Observational model: Case-Only
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        All patients who have consented to the Selective Dorsal Rhizotomy (SDR) procedure in one of
        the five Trusts who are commissioned to do SDR.