Review of Epidemiology and Service Use in Rare LTNCs (RESULT) study

Research Team: Professor Cath Sackley, Dr Tom Hoppitt, Sonal Shah
Start date: January 2009  End date: December 2010
Email: [email protected]


The RESULT study is looking at service provision for people with rare long-term neurological conditions. The conditions included in this study are Huntington’s Disease, motor neurone disease (amyotrophic lateral sclerosis), dominantly-inherited ataxias, multiple system atrophy, post polio syndrome, progressive supranuclear palsy, and charcot marie tooth disease. The National Service Framework for Long Term Neurological Conditions (NSF) has set out targets for services to improve care and treatment. However, little is known about the types of services people receive. This study aims to investigate the current provision of these services and how they need to change to meet the targets set out in the NSF.


  1. Actively engage with service users, carers and their organisations at all levels of the study. Use established techniques (such as Delphi and Nominal Group) to enable service users to identify their priorities for data collection and lead this activity. Ensure inclusion of 'hard to reach' groups to take account of the views of people from black and ethnic minority groups and people with rapidly degenerating health (such as MND). 
  2. Meet the aims and objectives of the LTNC research initiative and provide epidemiological data on rare progressive neurological conditions by the analysis and synthesis of existing data bases, existing data from clinical trials and other published literature.
  3. Provide information to underpin the NSF LTNC and commissioning as recommended in the NSF information strategy.
  4. Address issues of diversity by interrogating disease burden and resource use, particularly in black and ethnic minority populations and in addition identify areas of commonality
  5. Provide economic models that reflect disease progression at different stages.
  6. To collect primary data by survey and interview to establish social care and other societal costs and to enhance the quality of data collection among BME groups.
  7. Identify need for future studies

What we will do and how we will do it

There are 5 steps to the project and service users are involved in all aspects, Step 1 & 5 will be led by users.
1.Working withThe Dementias & Neurodegenerative Diseases Research Network (DeNDRoN) and the NHS Centre for Involvement, we will facilitate user-led consultations. Following feedback from DeNDRoN we will include specialist medical input to the process. In particular, we will include people from black and ethnic minorities and we have a local resource to help with that. In addition, Dr Paramjit Gill is very experienced in research including minority ethnic populations.   Opinions will be canvassed by email, together with interviews with people whose disease limits their participation (e.g. MND). The group will prioritise aspects of the course and consequences of neurological disease they would like to be captured in the study.
Parts 2-5 will include;
2. An extensive (systematic) review of the available data.
3. A review of existing GP records and hospital use data bases.   
4. Economic modelling of disease progression and cost at different stages.
5. Finally, service users will review the findings, lead dissemination and help design a programme of future studies

The results of the research

The study will provide data on the prevalence and incidence of rare progressive neurological disease and the use and cost of general and specialist services. It will provide detail of care management such as the timing of referral, drug history, access to rehabilitation and palliative services. This information will be available by population group, enabling breakdown by age, sex, ethnicity and locality and will provide an overview of any inequalities.
The information will be valuable to service users and their families, representative organisations, clinicians, commissioners and policy makers.
It will provide information identified by the NSF as important for the implementation and audit of the NSF LTNC, particularly QR2, QR4, QR7, QR9 and QR10.

Further information

Download a PowerPoint presentation Rare LTNC Oct 08 summarising our research plan.

Progress reports

Summer 2009 Update

University ethical approval has been obtained.  Also, NHS ethics was obtained pending development of social care data collection questionnaire (which will depend on outcome of Delphi completion which is due in September 2009).
The protocol for systematic review of epidemiology and symptomatic intervention has been completed, as has recruitment of Delphi service-user panel (n = 59). The panellists will identify service user priorities and are due to return the 1st round response.

A Protocol was submitted to ISAC and a revision requested.  It will be re-submitted in May 2009.

Service-users (patients, carers, health professionals, researchers) with neuro-conditions have been recruited to join the steering group advisory panel.

Winter 2009 Update

The Delphi process has been completed – there were a total of two rounds, the first round n=50 and the second round n=32.  There have been monthly meetings that have included service user input on the progress with the Delphi.

An application for THIN (The Health Improvement Network) data was approved and the research team received basic data training from THIN staff and statistical software training in SAS.  The data package was finally received on 16th October 2009

The interview schedule and questionnaires are in development and will have input from the reference panel.  Researchers are considering a number of strategies to engage with the BME community, e.g. visits to community groups, contact with a BME public health officer and help from staff experienced in accessing BME groups. Ethical approval was granted by South Staffordshire LREC (8th July 2009).

Summary of findings to date:

A systematic review of interventions to relieve symptoms of long-term neurological conditions has found following incidence/ prevalence statistics:

The Delphi process has highlighted a number of areas of care and management of the conditions that will be useful to get a complete picture of care from the database search. Highlighted areas of agreement and disagreement with aspects of health and social care will be used to develop the service user questionnaire. For example, various medical and allied health professionals that people might access were highlighted, such as cardiologist and hand therapist. This will help to provide a complete picture of the type of care people might access. A list of assistive aids that service users might use such as RADAR keys, the availability of accessible toilets, and a through-floor wheelchair lift were also mentioned. The 2nd round of the Delphi highlighted a number of areas of agreement.  For example, the panel agreed that multidisciplinary care was important for the management of the conditions, and that follow-up appointments should be available after diagnosis. These items will be followed up in the service user questionnaire to see whether this is happening in practice.

Summer 2010 update

The first draft of the second systematic review has now been completed.  A series of narrative papers, broken down by health condition, incorporating a review of interventions and work from other areas of the study, e.g. THIN (The Health Improvement Network) data, are currently in preparation.

With regard to our progress against targets for health economics and cost analyses;

We are also in the process of recruiting candidates for interview, with support from DENDrON and other charities. A questionnaire was developed with the reference panel and subsequently approved by ethics. It is now being finalised before being sent out.

Summary of findings to date

THIN data have revealed following results:





2.55 – 3.53

9.37 – 10.08


0.44 – 0.78

5.96 – 6.54


0.32 – 0.54

0.68 – 1.19


0.54 – 1.21

1.70 – 2.34


1.48 – 2.17

8.42 – 12.9


0.03 – 0.18

0.71 – 0.77

MND = Motor Neuron Disease
HD= Huntingdons Disease
MSA = Multiple System Atrophy
PSP= Progressive Supranuclear Palsy
CMT = Charcot Marie Tooth Disease
PP= Post Polio.


A poster, entitled ‘Using a primary care research database to estimate incidence and prevalence of long-term neurological conditions’ and another entitled ‘The Delphi technique to identify service user priorities in rare neurological conditions’ were presented at the at SWSAPC (South West region of the Society for Academic Primary Care) conference.




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