Recommendations for research
The Guideline Development Group (GDG) has made the following recommendations for research, based on its review of evidence, to improve NICE guidance and patient care in the future. The Guideline Development Group's full set of research recommendations is detailed in the full guideline.
What are the greatest inhibitors of functional ability in children and young people with upper motor neurone lesions?
Children and young people with upper motor neurone lesions may experience:
reduced muscle strength
selective muscle control
The relationships between these factors, and the extent to which the child or young person can develop or maintain functional ability, remain unclear. Prospective cohort studies, or large cross-sectional studies, are needed to explore the relationships between positive and negative effects of upper motor neurone lesions and to determine which factor is the greatest inhibitor of functional ability. The studies should incorporate classification of functional ability based on validated scales, such as the GMFCS.
What is the clinical and cost effectiveness of botulinum toxin type A when used routinely or according to clinical need in children and young people who are at GMFCS level I, II or III?
The GDG's recommendation to consider offering botulinum toxin type A to children and young people with focal spasticity of an upper or lower limb reflected available evidence relating to the safety and effectiveness of botulinum toxin type A. In making their recommendations, the GDG emphasised the importance of establishing individualised goals that justify the use of this potentially harmful toxin to treat spasticity. The cost of the procedure combined with the risk of side effects means that clear treatment goals that will positively influence the child or young person's life should be identified before offering this treatment. The evidence reviewed for the guideline provided limited support for botulinum toxin type A in terms of achieving clinically important goals (including those related to function), and this discouraged the GDG from making a strong recommendation to offer treatment with botulinum toxin type A to all children and young people who are at GMFCS level I, II or III. Further research is needed to evaluate the effectiveness of botulinum toxin type A in comparison with other treatment options, particularly when used over long time periods (for example, 10 years) and involving repeat injections, in this population of children and young people. Outcomes relating to improvements in gross motor function and participation in activities, and the psychological impacts of these factors, should be evaluated as part of the research.
What is the clinical and cost effectiveness of continuous pump-administered intrathecal baclofen compared with usual care in children and young people who are at GMFCS level IV or V?
The GDG's recommendation to consider offering continuous pump-administered intrathecal baclofen focused on children and young people in whom the use of appropriate non-invasive treatments did not relieve difficulties associated with spasticity (specifically pain or muscle spasms, posture or function, or ease of care). Such children and young people will typically be at GMFCS level IV or V. Further research is needed to evaluate the clinical and cost effectiveness of continuous pump-administered intrathecal baclofen compared with usual care in these children and young people. Relevant research designs include randomised controlled trials, prospective cohort studies and qualitative studies. The outcomes to be investigated as part of the research include: quality of life; reduction of pain; reduction of tone; acceptability and tolerability; participation or inclusion; and adverse effects and their association with any potential predisposing factors.
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?
The available evidence relating to selective dorsal rhizotomy suggests that the procedure results in some short- and medium-term improvements in motor function. The effects reported were not consistent across all studies nor sustained across all durations of follow-up investigated (6 to 24 months). The GDG considered that if the observed improvements could be maintained through to adult life then the outcomes of selective dorsal rhizotomy would be clinically important. Further research is urgently needed to evaluate long-term outcomes (including adverse effects) of selective dorsal rhizotomy followed by an intensive rehabilitation programme involving physical therapy (and prioritising targeted strength training) compared with physical therapy alone. The research could be conducted using a range of designs, including randomised controlled trials and audits of outcomes from procedures already performed. The research should focus on selective dorsal rhizotomy performed between the ages of 3 and 9 years in children who are at GMFCS level II or III (because these children are likely to benefit most from selective dorsal rhizotomy) and before the development of significant contractures at the ankles, knees and hips. The research should be coordinated through a multicentre research programme; use nationally agreed outcome measures (such as incidence of neurological impairment and spinal deformity, the need for additional operations, and assessment of disability, social inclusion and quality of life) and follow-up periods to facilitate national audit; and include assessment of the child's clinical condition before and after selective dorsal rhizotomy using the same formally validated assessment techniques. The full guideline includes further considerations relating to criteria for identifying children who could be included in the research, the timing of selective dorsal rhizotomy in relation to other treatments such as orthopaedic surgery, and information that should be given to children and their parents or carers to facilitate informed decision making about participation in research.