Care for Late Stage Parkinsonism

Parkinson*s disease (PD) affects approximately 1% of the population over 65 in
Western countries. Its prevalence is predicted to rise with the increase in the
ageing population. Health and social care needs will therefore also rise.
Health-care interventions for Parkinsonism are primarily directed towards
patients in the early stages of the disease, with a range of pharmacological
options and some non-pharmacological interventions including physiotherapy. As
the disease progresses, however, patients become more disabled and management
becomes more difficult. Complex drug treatment combinations often become
necessary to manage different motor and non-motor symptoms.Treatment options in
addition to oral antiparkinsonian medications include deep brain stimulation,
and two types of medication that are administered by continuous pump infusion
(apomorphine, levodopa/carbidopa intestinal gel) and require specialist input,
but these are suitable for and administered to only a minority of patients. In
the national guidelines for PD (e.g. NICE in the UK or guidelines of the German
Neurological Society) the treatment and care of this special population is
inadequately addressed. Many patients become immobile with declining response
to medications, falls, communication pro¬blems and high rates of non-motor
symptoms, such as behavioural and psychological symptoms (e.g. dementia,
depression, psychosis), autonomic failure, sleep disturbances and pain. Thus,
there is increased need for non-pharmacological intervention in addition to the
ongoing therapeutic adjustments of complex medication regimes. Mean time from
disease onset to the late disease stages in PD is estimated to be 14 years,
with considerable variation, but once milestones of this late stage are
reached, including falls, psychosis, or dementia, the prognosis is poor with an
average time to death of less than 5 years.

This project will investigate what the needs of patients with late stage
parkinsonism, their families and of their carers are, what services and support
they receive, and the extent to which their needs are met in different
health-care systems. This will be done through in-depth cross-sectional and
longitudinal assessments of a multicentred cohort study of patients, and
through interrogation of national or regional databases, where available. In
order to inform guidelines and provide the starting point for future randomised
controlled trials of therapeutic interventions in these patients, we will i)
undertake a review of the literature on effective treatments of late stage
Parkinsonism, ii) examine the outcome associated with different care models,
and iii) investigate the impact of a specialist assessment with management
suggestions, provision of guidance and access to telephone assistance on
patient-reported outcomes, carer burden and use of health-care resources. At
the conclusion of the study, we will produce a platform for assessment as well
as good practice guidelines for health and social care practitioners based on
the results.

Research questions are:
- What is the impact of late stage Parkinsonism on patients and their carers
and families and what are their medical and social needs, costs and use of
health-care resources?
- How appropriate and valid are the existing assessment tools in this
population?
- How do disability and disease severity milestones (psychosis, dementia,
falls, wheelchair-bound, institutionalisation) progress over 12 months in these
patients?
- What are the health-care and social determinants of outcome once disease
variables are accounted for?
- What is the impact of different health-care pathways?
- What are management strategies that have been shown to be effective in late
stage Parkinsonism?
- What is the impact of a specialist review with management recommendations,
provision of guidance and access to telephone assistance?

A longitudinal, multicentric, observational study in six European countries
with different health-care and social care models. In each country, 120
patients will be included, with two assessments (baseline and 6 months).

We will undertake an observational analysis of the data from the cohort study,
plus an open-label trial on the impact of specialist review with management
recommendations, guidance and availability of telephone assistance on outcome
at baseline and 8 weeks following the intervention. Eligible patients will be
randomised either receive recommendations by the research teams to their
primary health are team or continuing routine care management. There will be a
3:1 allocation to intervention with a quarter of randomly selected indivi¬duals
not receiving the intervention, except where is felt to be an urgent medical
need, e.g. contraindicated medications. We hypothesise that this specialist
input in the late stage of Parkinsonism provides better outcomes than standard
care.

The evaluation of the impact of the specialist review and management suggestion
takes place, 6 months after randomization.

The European protocol includes two additional assessments (at 12 and 18 months
after inclusion). Both assessments are optional and will not be done in the
Netherlands.

Management suggestions to the primary clinician involved in the care for the
patient, made by the senior physician/researcher at baseline following the
medical assessment, taking into account current and previous disease factors,
review of medications and current medical and social care arrangements. The
decision on implementation of any recommendations given will remain with the
primary health care team.

The suggestions may include recommendations on medication changes and referrals
for assessment by health-care services such as physiotherapist or other medical
specialties, and social care services. For physiotherapy, occupational therapy
and speech & language therapy guidelines are availble with indications for
referral plus evidence-based treatment suggestions. There will also be access
to telephone assistance by the study team over an 8 week period for patient,
carer and primary care physician.

Participation in the study begins by giving a written informed consent. Next,
two assessments are foreseen ( T1 in month 1 , T2 in month 6). These
assessments entail:

- Questionnaire for the patient, to be completed at home, 15 minutes each time,
2 times during the study period ( T1 , T2)
- Questionnaire for carers, to be completed at home, 15 minutes each time, 2
times during the study period ( T1 , T2)
- Medical assessment, consisting of an interview and observations by a
physician/researcher at the patient's home; 200-240 minutes each time, 2 times
during the study period ( T1 , T2)
- semi-structured interview with the patient and caregiver, at the patient's
home about care needs, 30 minutes; 1x during the study period (T1, optional).
- semi-structured interview with the patient and caregiver, at the patient's
home about received treatment recommendations; 15 minutes (T2, only for trial
participant in intervention group)

In particular, the medical assessment may be exhausting to the patient or
carer. Therefore it can be interrupted at any time if it is too heavy a burden
for the patient. The physician/researcher will later come back to finish the
assessment. In case the assessor is unsure about a particular assessment, the
patient will be asked for written consent to make a video recording. This video
will be presented to a second independent assessor for a second opinion. Both
assessors will work towards a consensus-based rating.

Depending on the recommendations, interventions can be implemented for those
patients assigned to the intervention group These interventions will only be
started after consent of the patient is obtained. The interventions include
both pharmacological interventions, and non -pharmacological interventions,
such as physical therapy, speech therapy or social work. The recommendations
aim to provide the best management for the patient.