Peninsula Cerebra Research Unit (PenCRU) for Childhood Disability Research 'What's the Evidence?' Compilation

What are ‘What’s the Evidence?’ reports?

The PenCRU ‘What’s the Evidence?’ reports are an ongoing series of short reports that seek to summarise what is known about the effectiveness of a particular treatment or therapy. These reports are a synthesis of existing research and are not new studies that we have carried out. They are written in response to queries from families about specific therapies and treatments.

We are told by families who work with us that looking for alternative treatments and therapies for their child is often a result of being given quite a bleak outlook from NHS clinicians, particularly soon after diagnosis. The idea of doing something to help their child is often better than accepting that ‘nothing can be done’. Whilst we understand this and are sympathetic, we are scientific researchers and believe it is important that families make informed decisions about whether to use any therapy or treatment. Therefore we look for evidence of effectiveness and seek to make families aware of what we find through the ‘What’s the Evidence?’ reports on the PenCRU website.

Where do we search for information?

When we produce a ‘What’s the Evidence?’ report we begin by searching the following databases:

The Cochrane Library
www.thecochranelibrary.com
The Cochrane Library is a collection of databases that contain different types of high-quality, independent evidence to inform healthcare decision-making. The evidence has been appraised using strict methods, so the conclusions are the best available.
TRIP database
www.tripdatabase.com
The TRIP Database is a search engine which allows clinicians to simultaneously search multiple sites providing the best available evidence.
Pubmed
www.ncbi.nlm.nih.gov/pubmed
PubMed comprises over 20 million citations for biomedical literature from MEDLINE, life science journals and online books.
NHS Evidence
www.evidence.nhs.uk
NHS Evidence provides free access to clinical and non-clinical information - local, regional, national and international. Information includes evidence, guidance and Government policy.
National Institute for Health & Clinical Excellence (NICE)
www.nice.org.uk
NICE is an independent organisation responsible for providing national guidance on promoting good health and preventing and treating ill health. The website gives access to all of the latest guidance.

As necessary we also contact providers of specific therapies, colleagues with an interest or expertise in the condition or therapy being queried, and any other relevant source of information.

Compilation of ‘What’s the Evidence?’ reports

At Cerebra’s invitation, this brief describes four topics that families have queried: Selective Dorsal Rhizotomy (SDR), Lycra Orthoses, Brainwave and Footsteps therapies, and the Scotson Technique.

Selective Dorsal Rhizotomy (SDR)

Selective Dorsal Rhizotomy is a neurosurgical procedure which can reduce spasticity of the lower limbs through selective division of sensory nerve fibre roots close to the spinal cord.

What were we asked?

Is Selective Dorsal Rhizotomy (SDR) an effective procedure for children with cerebral palsy?

What did we do?

We searched the usual databases for existing research.

We also contacted the Robert Jones & Agnes Hunt NHS Trust in Oswestry, and an orthopaedic surgeon with expertise in cerebral palsy not working at the Oswestry hospital.

What did we find?

Three randomised controlled trials have been published, and the results brought together in a meta-analysis.¹ A meta-analysis is a statistical strategy that is used to bring together the results of several studies into a single result. The studies showed, on average, a small improvement in movement ability in children who had SDR and physiotherapy compared to physiotherapy alone after one year follow up. These authors suggested that SDR is likely to be of most benefit to children between 3 and 8 years of age who walk with walking aids, external support or assistance (GMFCS levels III and IV). The meta-analysis was found to be of ‘reasonable quality’ by the Centre for Reviews and Dissemination at the University of York. This enhances the credibility and reliability of the conclusions of the study.² A protocol for conducting a Cochrane Review (high quality systematic review of the evidence) was identified but the report of the review has not been published.³

NICE have evaluated SDR and considered the findings of the meta-analysis, and also evidence from non-randomised clinical trials and case series. We have not repeated this work because it is available as part of their 2006 report.⁴

There are a number of potential short and/or long term adverse outcomes from SDR highlighted in the 2006 NICE report; these are pain, bowel and bladder functioning, and spinal deformity. Nevertheless, the NICE guidance states that “…the safety of selective dorsal rhizotomy (SDR) for spasticity in cerebral palsy appears adequate; however, there is evidence of only limited efficacy (effectiveness). Therefore, the procedure should not be used without special arrangements for consent and for audit or research.” ⁴

NICE published revised guidance in December 2010 which states “Current evidence on selective dorsal rhizotomy for spasticity in cerebral palsy shows that there is a risk of serious but wellrecognised complications. The evidence on efficacy is adequate. Therefore this procedure may be used provided that normal arrangements are in place for clinical governance and audit.”

Experience of SDR in the UK has largely been amongst patients treated in Oswestry.⁵ This centre has reported their experience and patient outcomes in a peer-reviewed publication. A strict process for selecting patients appropriate for SDR is described, and only half of the children originally referred for assessment actually had SDR. Walking ability and gait improved among the selected patients.⁵

Recent publications have included further follow up of patients who had SDR, and suggest that walking ability is maintained in the long term. ^{6, 7} Other studies have examined factors associated with better outcomes, identifying some associations between movement ability and MRI brain scan findings,8 and to a lesser extent measures of spasticity, strength and gait.⁹

What do we think?

There has been considerable evaluation of the effectiveness of SDR for children with cerebral palsy. Overall the evidence suggests a beneficial effect on reduction of spasticity and a small or moderate improvement in movement ability in a carefully selected group of patients. However, controversy remains about precisely which children are likely to benefit.

When considering children for the procedure, surgeons in the UK centre appear to prefer that children are slightly older than in the USA. The hospital in Oswestry does not usually consider using SDR with children under 5 years so that their mobility and gait have matured. However surgeons in the USA appear willing to consider using SDR with children as young as 2 years old. It is important to note that SDR does not resolve fixed lower limb deformities. When children are found to have fixed deformities, consideration will be given to addressing these with orthopaedic surgery. In principle, permanently reducing lower limb spasticity with SDR in younger children might prevent the development of deformity and reduce the need for orthopaedic surgery as children get older. This has been studied, though the evidence is not conclusive.¹⁰

The authors of the meta-analysis suggested the procedure was most likely to benefit children in GMFCS levels III and IV (who need mobility aids to walk); however more recent publications include some children in GMFCS levels I and II (who may not use walking aids).

There appears to be a difference in the proportion of nerves which are severed during the procedure, with more extensive surgery being carried out in the USA. The more nerves that are severed then the greater the consequent reduction in spasticity.

The revised guidance issued by NICE in December 2010 means that SDR can be used as a treatment in the NHS subject to the necessary arrangements being in place. We heard recently from a parent of a child who was being considered for the procedure at a hospital in Bristol.

Our recommendations

The answer to the question we were posed is not straightforward. SDR appears to be somewhat effective for a subgroup of children with cerebral palsy whose mobility is limited by excessive spasticity. Improvements in movement ability may be achieved following the procedure by reducing the spasticity. When improvements occur they appear to be maintained in the medium to long term.

However, the characteristics of children who achieve the best results are not well defined, and the outcomes are occasionally disappointing. Further research is required to ascertain which children are most likely to benefit from SDR, especially in terms of age and severity of movement disability.

Any families wishing to find out more about SDR should read the guidance published by NICE on their website.⁴

References

McLaughlin J, Bjornson K, Temkin N, Steinbok P, Wright V, Reiner A, Roberts T, Drake J, O’Donnell M, Rosenbaum P, Barber T, Ferrel A. (2002) Selective dorsal rhizotomy: meta-analysis of three randomized controlled trials. Developmental Medicine and Child Neurology 44(1):17-25. http://www3.interscience.wiley.com/cgi-bin/fulltext/118939714/PDFSTART.
Database of Abstracts of Reviews of Effects, Centre for Reviews and Dissemination, University of York. (2008) Selective dorsal rhizotomy: meta-analysis of three randomized controlled trials. www.crd.york.ac.uk/crdweb/ShowRecord.asp?View=Full&ID=12002000262.
Narayanan UG, Howard WG. (2001) Selective dorsal rhizotomy in the management of children with spastic cerebral palsy. Cochrane Review Protocol. http://mrw.interscience.wiley.com/cochrane/clsysrev/articles/CD003360/frame.html .
National Institute for Health & Clinical Excellence (NICE) (2006) Selective dorsal rhizotomy for spasticity in cerebral palsy. Guidance updated 2010 http://guidance.nice.org.uk/IPG373/Guidance/pdf/English. 2006 report www.nice.org.uk/nicemedia/live/11220/52083/52083.pdf. All relevant NICE documents www.nice.org.uk/IPG195.
Cole GF, Farmer SE, Roberts A, Stewart C, Patrick JH. (2007) Selective dorsal rhizotomy for children with cerebral palsy: the Oswestry experience. Archives of Diseases in Childhood. 92(9):781-5. http://adc.bmj.com/content/early/2007/05/02/adc.2006.111559.short.
Nordmark E, Josenby AL, Lagergren J, Andersson G, Strömblad LG, Westbom L. (2008) Long-term outcomes five years after selective dorsal rhizotomy. BMC Pediatrics 14;8:54. http://www.biomedcentral.com/1471-2431/8/54.
Langerak NG, Lamberts RP, Fieggen AG, Peter JC, van der Merwe L, Peacock WJ, Vaughan CL. (2008) A prospective gait analysis study in patients with diplegic cerebral palsy 20 years after selective dorsal rhizotomy. Journal of Neurosurgical Pediatrics. 1(3):180-6. http://thejns.org/doi/full/10.3171/PED/2008/1/3/180. Also read the commentary http://thejns.org/doi/full/10.3171/PED/2008/1/3/178.
Grunt S, Becher JG, van Schie P, van Ouwerkerk WJ, Ahmadi M, Vermeulen RJ. Preoperative MRI findings and functional outcome after selective dorsal rhizotomy in children with bilateral spasticity. Childs Nerv Syst. 2009 Oct 13. www.ncbi.nlm.nih.gov/pmc/articles/PMC2800177.
Engsberg JR, Ross SA, Collins DR, Park TS. Predicting functional change from preintervention measures in selective dorsal rhizotomy. J Neurosurg. 2007 Apr;106(4 Suppl):282-7. www.ncbi.nlm.nih.gov/pmc/articles/PMC2678243/pdf/nihms52698.pdf.
Chicoine MR, Park TS, Kaufman BA. Selective dorsal rhizotomy and rates of orthopedic surgery in children with spastic cerebral palsy. J Neurosurg. 1997 Jan;86(1):34-9. www.ncbi.nlm.nih.gov/pubmed/8988079.

Lycra Orthoses

Lycra orthoses are a form of support which may benefit people with conditions affecting movement and posture, such as cerebral palsy.

What were we asked?

A parent of a young child with hemiplegia asked whether there was any evidence that Lycra suits are effective, as they had heard conflicting reports. They were concerned that it may weaken muscles, and also asked whether there is any ongoing benefit once suit is no longer worn.

What did we do?

We transformed the query into a question: ‘Do Lycra orthoses (splints) improve function and movement ability in children with hemiplegia, or other types of cerebral palsy?’ Then we searched the usual databases for existing research.

What did we find

Although Lycra orthoses have been used for around 10 years, there are very few research studies that have looked at the effectiveness of these garments. There are some short-term before and after studies, but long term effectiveness has not been evaluated. There have been no randomised controlled trials of the effectiveness of Lycra garments. Currently, there are no NICE guidelines on the use of Lycra splinting.

There are several ‘review articles’ that have tried to bring together the evidence from smaller studies.^1,2,3 These three reviews include many of the same studies and surmise that current evidence suggests that wearing Lycra garments may improve stability, function and range of movement in the short term for some children with cerebral palsy but that the evidence is limited and difficult to compare. There were lots of things about the studies that made it difficult for them to draw conclusions. The studies had very few children participating (the number of children ranged from two to 24 depending on the study). The research designs lack adequate comparison to the Lycra orthoses e.g. making the same observations on similar children that don’t have Lycra garments to see what we would expect to happen anyway. The studies also differed in the way that they measured whether Lycra orthoses were having an effect. Some studies created their own measure, which means they can’t easily be compared to the other studies.

A further review paper ⁴concluded that the lack of randomised controlled trial data means that there is currently no solid evidence to support the use of Lycra splints. While some children do seem to benefit from using Lycra orthoses, some families report difficulties with the garments for a number of reasons: they can be hard to get on and off; children may feel hot and restricted in them; it can feel difficult to breath and toileting may be a consideration with full body suits.^2,5, In some of the studies, the discomfort and inconvenience reported were bad enough that families stopped using the garments all together or as much as was recommended and some parents reported that the practicalities of using the garment would put them off using Lycra garments again. There have not been any studies that have looked at whether Lycra garments enable children to participate more in a child’s typical activities.

What do we think?

Because there is little evidence to compare, it is very difficult to say for sure whether children will benefit from using Lycra garments, how they might benefit, which children are most likely to benefit, and whether any benefits are sustained in the long term. The lack of long-term studies makes it impossible to provide dependable advice about whether there is a risk of weakened muscles or any lasting benefit once a garment is no longer worn. It is important to remember that this doesn’t mean that Lycra orthoses don’t work. It means that there haven’t been enough well conducted studies of the same garment in groups of children with the same condition for us to be able to say that the evidence clearly shows that they do work. The people that wrote the review articles all said that randomised controlled trials were needed to properly assess the benefit of Lycra garments.

References

J.E. Coghill and D.E.Simkiss. Do Lycra garments improve function and movement in children with cerebral palsy? Arch Dis Child 2010; 95: 393-395.
National Horizon Scanning Centre. Lycra garments for cerebral palsy and movement disorders. Birmingham: National Horizon Scanning Centre. 2002. www.haps.bham.ac.uk/publichealth/horizon/outputs/documents/2002/Lycragarments.pdf Accessed 16 July 2010.
J. Attard and S Rithalia. A review of the use of Lycra pressure orthoses for children with cerebral palsy. Int J Ther Rehabil. 2004; 11: 120-126.
Blackmore A.M. Garbellini SA Buttigieg PJ Wells B. A systematic review of the effects of soft splinting on upper limb function in people with cerebral palsy: an AACPMD Evidence Report. 2006. www.aacpdm.org/membership/outcome/resources/Softsplintingreview.pdf Accessed 3 August 2010.
NHS Quality Improvement Scotland. Dynamic Lycra splinting for children with cerebral palsy. NHS Quality Improvement Scotland Evidence Note #11. 2005. www.nhshealthquality.org/nhsqis/files/EN11%20Final.pdf Accessed 16 July 2010.

Brainwave and Footsteps

Brainwave and Footsteps each provide intensive physiotherapy based treatment programmes. Brainwave devises a programme to be carried out at home, and Footsteps provide their therapy within their premises in Oxford.

What were we asked?

Parents asked whether there was any research that therapies provided by Brainwave and Footsteps are effective, especially as they had heard they were expensive. These are two separate services that offer therapy and advice outside of the NHS.

What did we do?

We searched the usual databases for existing research. We also contacted the Brainwave and Footsteps centres and asked about their programmes, any differences with NHS therapy provision, and whether there was evidence to support the improvements that they claim; we also asked the cost and time required for assessment and therapy.

What did we find

The programmes provided by these organisations appear to involve ‘mostly’ mainstream rather than ‘alternative’ interventions. Both organisations operate as registered UK charities and herefore seek to overcome any financial barriers to accessing their treatments.

Brainwave are a multidisciplinary team of health and education professionals who assess children with a variety of neurological and developmental conditions, and give advice on a therapy programme which parents are then encouraged to carry out at home. Brainwave costs £450 for an initial assessment and the advice and any equipment; follow up evaluations every 4 months are recommended at a further cost of £250.

Footsteps offer hands-on physiotherapy, at their premises in Oxfordshire, for children and young adults with neurological conditions. Footsteps use apparatus for simulating standing as part of their therapy, the so-called spider, which distinguishes their treatment from conventional physiotherapy. We are not aware of any scientific study of the effectiveness of this apparatus. The cost of therapy at Footsteps is charged at a rate of £40 per hour; three week intensive programmes are offered at the same rate.

We are not aware of any scientific evidence of the effectiveness of either programme to improve children’s functioning.

What do we think?

We think that Brainwave and Footsteps seek to offer a higher ‘dose’ of therapy than might be provided by the NHS. Brainwave does this by encouraging parents to carry out therapy at home; while therapists at Footsteps deliver their therapy directly. Does the dose of therapy make a difference? Well, the optimum dosage of therapy is not really known, and would best be evaluated in well designed research study.

Parents want the best for their children, and families with disabled children may want to seek additional therapy even without knowing if it will help. Similarly, some parents employ the services of private physiotherapists not allied to these organisations. Children affected by neurological conditions are often late developers; typically they are developing all the time.Therefore it is difficult to determine whether any improvement is above what would be expected to happen in due course with less therapy or even no treatment.

Our recommendations

A controlled clinical trial is required to assess whether the intensive therapy programmes suggested by Brainwave, carried out by families at home, improve children’s functioning and whether any benefit is maintained over the long term.

A controlled clinical trial is required to assess the contribution of the ‘spider’ standing apparatus to any improvements gained through intensive physiotherapy provided by Footsteps.

A controlled clinical trial is required to assess whether increasing the frequency and/or intensity of physical, occupational and speech and language therapies improves children’s functioning and any benefit is maintained over the long term.

Lastly, we think children’s lives are about more than ‘treatment and therapy’ and we advocate that sufficient time should be given to all kinds of recreational activities.

Further information

We have provided links to these organisations for reference only; as can be seen from our comments, in no way do we endorse the products offered by these organisations:

If you are seeking private physiotherapy for your child then this link may be helpful:

www.apcp.org.uk/InformationforParents/tabid/69/Default.aspx

The Scotson Technique

Scotson therapy involves parents learning to apply light pressure on the muscles that are involved in breathing and posture. The techniques are applied in the belief that this will stimulate breathing, posture and function.

What were we asked?

A parent asked if there was any evidence that the Scotson technique is effective. The Scotson technique is a kind of touch therapy available from the Advance Centre.

What did we do?

We searched the usual databases for existing research.

We also contacted the Advance Centre which provides Scotson therapy; we spoke with Linda Scotson directly. We were referred to a study conducted by the University of Bradford that was sponsored by Advance; the report of that study is available online. ¹

What did we find

Scotson believes in a link between breathing and posture that adversely affects children’s postural and functional development.

The theory by which the Scotson technique might work has not been established. The Bradford study considered ways in which the theory behind the Scotson treatment might be related to established physiological principles, but many of the assumptions remain to be substantiated. The Bradford study followed a large number of children who were receiving Scotson therapy. Changes in body size measurements seen in these children are unlikely to reflect any functional importance, nor be attributed to development and maturation, and only 3 of the 18 changes in body measurement were reported to be statistically significant.

Our recommendations

There is no scientific evidence that Scotson therapy is effective in improving children’s breathing, posture, functioning or health. We are highly sceptical about Scotson therapy and the theory behind how it is supposed to work. We have major concerns with the claim that improvements can be achieved with ‘all’ children, even though they may have quite different conditions and diagnoses.

Further information

We have provided a link to this organisation for reference only; as can be seen from our comments, in no way do we endorse the products offered by this organisation.

www.scotsontechnique.com

References

Rachael Sharples (2005) The Scotson technique. An exploratory study to establish a theoretical base for the technique and investigate any potential effects. University of Bradford www.scotsontechnique.com/Bradford_Report_Full.pdf

Conclusions

These four ‘What’s the Evidence?’ reports highlight the range of treatments that families are interested in and that PenCRU has been asked about. Treatments include those offered on the NHS, privately, and also complementary and alternative therapies. An important consideration when deciding whether to use any treatment should be whether there is any evidence that a treatment works. It is also sometimes relevant to consider whether there is any evidence to suggest how often the treatment should be used in order to achieve the desired outcome. Therefore it is always important to look for evidence of high quality research so that you make informed decisions about the right choice for your child.

From the reports above we can conclude the following. SDR has been subject to high quality research over 20 years which has led to NICE guidelines which recommend that the surgery may be offered subject to defined resources being available. SDR is a treatment available through the NHS. Lycra-based orthoses are also available on the NHS in some but not all areas; this is because there is little evidence that Lycra-based orthoses are effective and knowledge about which children might benefit is lacking. There is a need for a long term study which will clarify the effectiveness of these garments to inform decision-making, but this does not necessarily mean that they don’t work.

As far as we are aware there has been no research evaluating the effectiveness of either the Brainwave or Footsteps programmes. These programmes have emerged as commercial/ charitable enterprises, which are quite expensive when families do not obtain charitable support. Essentially these therapies appear to be intensive doses of mainstream therapies provided by the NHS. Note that a key part of the therapy offered by Footsteps involves novel equipment that is not used in the NHS, as the effectiveness of this equipment remains unproven.

There is no evidence regarding the effectiveness of the Scotson Technique, and from a scientific perspective it is hard to understand the theory behind how it is claimed to work.

Who we are

Staff at PenCRU (Peninsula Cerebra Research Unit for Childhood Disability) work in partnership with parents and carers of children with additional needs to ask and answer questions about treatments and services related to their children. We are also developing ways to involve children in PenCRU’s activities.

PenCRU was founded to help establish what treatments and therapies improve the health and wellbeing of children with disabilities and their families. We respond to questions from families about therapies and health services for children and young people with brain-related neurological conditions, and provide summaries of evidence to help families make decisions. Where the evidence is lacking, PenCRU works with families to prioritise research topics and seek funding to conduct clinical trials to assess the effectiveness of interventions. PenCRU is interested in outcomes that families tell us are important, such as function, social inclusion and participation, and quality of life.