2 Research recommendations

2.1 A key worker approach for children and young people with autism and their families

What is the value of a key worker approach (defined by protocol and delivered in addition to usual care) for children and young people with autism in terms of parental satisfaction, functioning and stress and child psychopathology?

Why this is important

Autism is well characterised as a chronic disorder with lifelong disability in some individuals, yet the current health management structure is usually organised around single episodes of care. The theory and practice of management of chronic illness, as well as widely expressed service-user opinion, indicate that a chronic care model for the organisation of autism services could be appropriate and cost effective.

A key worker approach for children and young people with autism and their families should be formally evaluated in a randomised controlled trial (RCT) reporting short- and medium-term outcomes (including cost-effectiveness) with a follow‑up of at least 6 months and again at 12 months. The outcomes (parental satisfaction, functioning and stress and child psychopathology) should be assessed by structured clinical interviews, parent- and self-reports using validated questionnaires and objective measures of behaviour. The study needs to be large enough to determine the presence of clinically important effects, and mediators and moderators (in particular the child or young person's age) should be investigated.

2.2 Managing behaviour that challenges in children and young people with autism

Is a group-based parent training intervention for parents or carers of children and young people with autism clinically and cost effective in reducing early and emerging behaviour that challenges in the short- and medium-term compared with treatment as usual?

Why this is important

Behaviour that challenges is common in children and young people with autism but many are referred only when the behaviour has become severely impairing, they pose a threat to themselves or others, or everyday life has broken down. By this time, behavioural interventions may be difficult or impossible and antipsychotic medication is used despite it being symptomatic in its benefits, having long-term adverse effects and behavioural problems typically recurring after use.

A group-based parent training intervention (such as educating parents to identify triggers and patterns of reinforcement) should be evaluated using an RCT. Primary outcomes should be short- and medium-term reduction in behaviour that challenges. Secondary outcomes should include parental and sibling stress, quality of life and the child or young person's adaptive function. The medium-term use of medication should also be assessed. Cost effectiveness should encompass a wide range of services, such as additional educational support and social services, and health service use by families.

2.3 Managing sleep problems in children with autism

Is a sleep hygiene intervention or melatonin clinically and cost effective in the management of sleep onset, night waking and reduced total sleep in children (aged 4–10 years) with autism?

Why this is important

Sleep problems are common in children and young people with autism and have a significant negative impact on them and their parents. However, studies of melatonin have used different groups and preparations of melatonin precluding meta-analysis.

The intervention should be evaluated in an RCT in 3 stages: (1) recording sleep onset, night waking and total sleep time over 3 months using actigraphy and a parent-completed diary; (2) for those with a sleep problem, random allocation to sleep hygiene by booklet or professional contact; (3) for those with persistent sleep problems after 3 months, random allocation to prolonged-release melatonin or placebo; after a further 3 months, those on placebo would be offered melatonin.

It should report primary and secondary outcomes followed up at 12 months for all participants. Primary outcomes should include increased total sleep time and decreased night waking. Secondary outcomes should include improved sleep onset, a change in Aberrant Behaviour Checklist measures of behaviour that challenges, and improvement in parental stress index and satisfaction and the child's cognitive function.

2.4 Treating comorbid anxiety in children and young people with autism

What is the comparative clinical and cost effectiveness of pharmacological and psychosocial interventions for anxiety disorders in children and young people with autism?

Why this is important

Early trials of CBT for anxiety in children and young people with autism have been promising but have methodological shortcomings. Furthermore, the common pharmacological approaches have not been evaluated in this population.

A parallel-arm RCT should compare pharmacological and psychosocial interventions with placebo in children and young people with autism and an anxiety disorder. Pharmacological treatment should be with a selective serotonin reuptake inhibitor (SSRI) and dosing should follow research in typically developing children but with the option of evaluating outcomes at lower doses. The SSRI should be blinded with an identical placebo and an 'attention' or other psychosocial control group. The psychosocial intervention should be manualised and based on cognitive behavioural approaches shown to be effective in previous trials. The sample should cover the full age and intellectual range of children and young people and the size powered to deliver precise effect size estimates for both active arms.

Primary outcome measures should be reduction in anxiety symptoms by parent report. Secondary outcomes may include self- and teacher-report, blinded measures such as heart rate and skin conductance, patient satisfaction, changes in adaptive function, quality of life and disruptive behaviour. Adverse effects should be evaluated and an economic evaluation included.

2.5 Teacher-, parent- and peer-mediated psychosocial interventions in pre‑school children with autism

Are comprehensive early interventions that combine multiple elements and are delivered by parents and teachers (for example, the Learning Experiences – an Alternative Program for Preschoolers and their Parents [LEAP] model) effective in managing the core symptoms of autism and coexisting difficulties (such as adaptive behaviour and developmental skills) in pre‑school children?

Why this is important

Many children with autism are diagnosed in the pre‑school period when service provision is advice and support to parents and professionals in nursery or early years educational settings. There is evidence from one moderate-sized trial that adequately supervised comprehensive programmes can help manage the core symptoms of autism and coexisting difficulties. However, the quality of the trial was low.

The research programme should be in 4 stages:

1. Develop a manualised programme suitable to UK public service settings (health services, early years education, and so on).

2. Test its feasibility and acceptability in pilot trials with blinded assessment of outcome.

3. Formally evaluate the outcomes on core symptoms of autism and coexisting difficulties in a large-scale trial, including health economic analysis.

4. Conduct a series of smaller trials to determine the elements, length and intensity required to ensure effectiveness of the programme, as well as longer-term outcomes.

  • National Institute for Health and Care Excellence (NICE)