NumberResearch recommendationSee section
Inhibitors of functional ability4
1What are the greatest inhibitors of functional ability in children and young people with upper motor neurone lesions?4
Why this is important
Children and young people with upper motor neurone lesions may experience:
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.
Postural management4
2What is the optimal postural management programme using a standing frame in children aged 1–3 years?4
Why this is important
Children who are at GMFCS level IV or V may benefit from using a standing frame as part of a postural management programme. Clinical benefits might include improved weight bearing and walking and, as a result, reduced hip migration. Postural management programmes involving the use of standing frames are part of established clinical practice. However, the individual elements that optimise the effectiveness of such programmes merit further research. The research should compare the effectiveness of postural management programmes that incorporate different durations and timings of standing frame use. For example, what is the effectiveness of 1 hour per day in a single session compared with two sessions of 30 minutes per day? The research should be conducted in children aged 1–3 years. These children are likely to benefit the most from using standing frames (in terms of developing well-formed femoral heads and acetabulums) and they should find the use of standing frames acceptable (because they are lighter than older children and they do not have severe contractures).
Botulinum toxin type A7
115What 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?7
Why this is important
The Guideline Development Group's (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.
Intrathecal baclofen8
21What 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?8
Why this is important
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.
Selective dorsal rhizotomy10
25Does 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?10
Why this is important
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 (6-24 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 and this would be a cost effective treatment option. 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 with spasticity 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 following criteria should help to identify children who could be included in the research: abnormal tone (pure spasticity), good leg muscle strength, straight legs and minimal muscle shortening, good selective motor control in the legs, good cognitive skills, and not being overweight. Abnormal tone that is predominantly dystonia, and severe scoliosis or hip dislocation, should form part of the exclusion criteria. 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; consider the timing of selective dorsal rhizotomy in relation to orthopaedic surgery if the child has muscle shortening or torsional abnormalities; consider the involvement of the child, their parents, carers or other family members, and members of the local multidisciplinary child development team in the rehabilitation programme after discharge from hospital; monitor the child's clinical condition regularly until they are fully grown (to detect and manage weight gain and orthopaedic and spinal complications). The following information should be given to children and their parents or carers to facilitate informed decision making about participation in research: selective dorsal rhizotomy is irreversible; there is a risk of serious temporary or permanent postoperative complications (such as deterioration in walking ability or bladder function) and later complications such as spinal deformity; prolonged physiotherapy and aftercare will be needed; additional surgery may be needed; subsequent to selective dorsal rhizotomy epidural anaesthesia will not be possible (for example, during additional surgery or childbirth); the evidence already available in relation to selective dorsal rhizotomy is based on studies involving small numbers of children, and there is currently no evidence from which to assess long-term outcomes (those experienced more than 24 months after performing selective dorsal rhizotomy, and preferably into adult life); confounding factors for long-term outcomes could include the natural history of the condition (for example, the child's condition might deteriorate over time regardless of whether or not selective dorsal rhizotomy is performed).

From: 1, Guideline summary

Cover of Spasticity in Children and Young People with Non-Progressive Brain Disorders
Spasticity in Children and Young People with Non-Progressive Brain Disorders: Management of Spasticity and Co-Existing Motor Disorders and Their Early Musculoskeletal Complications.
NICE Clinical Guidelines, No. 145.
National Collaborating Centre for Women's and Children's Health (UK).
London: RCOG Press; 2012 Jul.
Copyright © 2012, National Collaborating Centre for Women's and Children's Health.

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