Recommendations for research

The Guideline Committee has made the following recommendations for research. The Committee's full set of research recommendations is detailed in the full guideline.

1 Organisation of care

Is a network‑based model as effective as a clinic‑based model to deliver multidisciplinary care to people with motor neurone disease (MND)?

Why this is important

Multidisciplinary care improves survival in patients with MND. The evidence is drawn from models of multidisciplinary care that use a clinic based approach. However there are other models of care delivery in practice including care networks. Often these alternative models of care have arisen out of necessity in large geographical regions with low density populations. These alternative models may have similar survival advantages to patients with MND and this needs to be established.

2 Cognitive assessment

What is the impact of assessing for cognitive and behaviour change in people with MND on clinical practice, the person and their family and carers? Does repeated assessment provide more benefit than assessment at a single point at diagnosis?

Why this is important

Clinic‑based and population‑based studies demonstrate that up to 15% of people with MND have frontotemporal dementia. A further third of people with MND have changes in behaviour and cognition. These impairments are present at diagnosis. Their course during the disease has shown varying patterns between studies although several studies have shown that cognitive and behavioural impairments predict poorer survival and increased carer burden. A randomised controlled trial is needed to assess whether formal assessment at diagnosis and/or repeated assessment improves clinical practice, subsequent care of the person and quality of life for the person, their family and carers.

3 Prognostic tools

Is the ALS Prognostic Index an accurate predictor of survival in people with MND under NHS care in England and/or Wales?

Why this is important

Accurate predictions of survival in people with MND would be of great use to clinicians and to the person with MND, their family and carers. Accurate predictions would enable people with MND to be clearer about their prognosis, make plans for the rest of their life and have a well‑prepared and dignified transition into the end of life phase. Family members would similarly benefit in being more aware of the likely progression and prepare themselves for the death of their loved one.

Accurate predictions of survival would enable professionals to create and deliver more effective management and care plans and access services when it is most appropriate, for example specialist palliative care.

The ALS Prognostic Index (ALS‑PI) was developed in a cohort of people with ALS in the Republic of Ireland and externally validated in a cohort in Italy. However, it has not been validated in people with ALS, primary lateral sclerosis or progressive muscular atrophy in the NHS in England or Wales. The tool needs to be validated in a UK population using a simplified measure of executive function.

4 Saliva

How is excessive drooling of saliva (sialorrhoea) managed in people with MND?

Why this is important

Sialorrhoea affects up to 50% of people with MND and in 42% of these individuals the symptom is poorly controlled. There is no evidence base for clinicians to make decisions with regards to the treatment options available. Antimuscarinics are used first‑line but there is no evidence to inform which antimuscarinic and at what dose. Botulinum toxin is used second- or third‑line although there is little evidence to guide dosing, which salivary glands to inject and which type of botulinum toxin to use. Currently there is no baseline information about how specialists are using these treatments and this information is required to inform comparative studies.

5 Nutrition

Does a high calorific diet prolong survival of people with MND if initiated following diagnosis or following initiation of feeding using a gastrostomy?

Why this is important

There is little specific guidance on the optimal calorie intake for people with MND. There is growing evidence that people with MND have a hypercatabolic state and have high energy requirements. A large cohort study in the UK has demonstrated that nearly half of people continue to lose weight following gastrostomy and most show no improvement in their weight. A small study has demonstrated that high fat and high carbohydrate feeding may prolong survival in gastrostomy‑fed people. A larger randomised trial is needed to inform clinical practice.

6 Augmentative and alternative communication

What is the current pattern of provision and use of augmentative and alternative communication (AAC) by people with MND in England?

Why this is important

Appropriate AAC equipment can have a significant effect on quality of life for people with MND. While the NHS has a responsibility to provide equipment and ongoing support in its use, there are no reliable data on the types of equipment found most useful at different stages of the disease process, or the number of people with MND who may benefit from AAC. A prospective census study of people with MND presenting with early onset of speech problems is needed to establish the current baseline provision and needs of this population and how best to utilise AAC equipment. The programme will begin with the collection and analysis of basic data. It will then progress to patient‑related outcomes.

  • National Institute for Health and Care Excellence (NICE)