Rachel Burman

Is Short-Term Palliative Care Cost-Effective in Multiple Sclerosis? A Randomized Phase II Trial

CONTEXT Palliative care is being advocated for noncancer patients but needs evidence of effectiveness and cost-effectiveness. OBJECTIVE We evaluated the cost-effectiveness of a new palliative care service for people with multiple sclerosis (MS). METHODS We used a randomized fast-track Phase II controlled trial. Patients in South East London who were severely affected by MS were referred by clinicians to the trial. After baseline interview, patients were randomly allocated to either a multiprofessional palliative care team (PCT) immediately (fast track) or the control care group who continued best usual care for three months and then were offered the PCT. Data were collected at baseline, 6, 12, 18, and 26 weeks on use of services, patient symptoms, other outcomes, and caregiver burden. RESULTS Fifty-two patients were randomized: 25 fast track and 21 control patients completed the trial. There was a high level of disability, and mean Expanded Disability Status Scale score was 7.7 (median 8, standard deviation 1.0). At 12 weeks, caregiver burden was 4.47 points lower (95% confidence interval [CI]: 1.05-7.89) in the fast track compared to the control group. Mean service costs, including inpatient care and informal care, over the 0-12-week follow-up were pound1,789 lower for the fast-track group (bootstrapped 95% CI: - pound5,224 to pound1,902). There was a trend toward lower community costs in the fast-track group and no differences in costs to informal caregivers. CONCLUSIONS The trial suggests that short-term palliative care for people severely affected by MS and their caregivers will be cost-effective and warrants further study. The fast-track trial design could be used to assess this.

A proposed staging system for amyotrophic lateral sclerosis

Amyotrophic lateral sclerosis is a neurodegenerative disorder characterized by progressive loss of upper and lower motor neurons, with a median survival of 2–3 years. Although various phenotypic and research diagnostic classification systems exist and several prognostic models have been generated, there is no staging system. Staging criteria for amyotrophic lateral sclerosis would help to provide a universal and objective measure of disease progression with benefits for patient care, resource allocation, research classifications and clinical trial design. We therefore sought to define easily identified clinical milestones that could be shown to occur at specific points in the disease course, reflect disease progression and impact prognosis and treatment. A tertiary referral centre clinical database was analysed, consisting of 1471 patients with amyotrophic lateral sclerosis seen between 1993 and 2007. Milestones were defined as symptom onset (functional involvement by weakness, wasting, spasticity, dysarthria or dysphagia of one central nervous system region defined as bulbar, upper limb, lower limb or diaphragmatic), diagnosis, functional involvement of a second region, functional involvement of a third region, needing gastrostomy and non-invasive ventilation. Milestone timings were standardized as proportions of time elapsed through the disease course using information from patients who had died by dividing time to a milestone by disease duration. Milestones occurred at predictable proportions of the disease course. Diagnosis occurred at 35% through the disease course, involvement of a second region at 38%, a third region at 61%, need for gastrostomy at 77% and need for non-invasive ventilation at 80%. We therefore propose a simple staging system for amyotrophic lateral sclerosis. Stage 1: symptom onset (involvement of first region); Stage 2A: diagnosis; Stage 2B: involvement of second region; Stage 3: involvement of third region; Stage 4A: need for gastrostomy; and Stage 4B: need for non-invasive ventilation. Validation of this staging system will require further studies in other populations, in population registers and in other clinic databases. The standardized times to milestones may well vary between different studies and populations, although the stages themselves and their meanings are likely to remain unchanged.

A prospective survey of the use of dexamethasone on a palliative care unit

One hundred and six consecutive patients started on glucocorticosteroids (steroids) according to a defined prescription policy were surveyed each week to document the indications for use, any beneficial effect, any toxicity incurred and the reason for stopping. All patients had advanced malignant disease and survived for a median of 40.5 days (range 1–398+ days) from the start of steroid treatment. Fifty-seven per cent of patients completed three or more assessments. The most common specific indications for starting steroids were spinal cord compression, cerebral metastases, lymphangitis carcinomatosa and intestinal obstruction. The most common non-specific indications were anorexia, nausea, low mood, pain and vomiting. The median duration of steroid use was 21.5 days (range 1–89 days). The most common reason for the discontinuation of steroids was death or deteriorating condition. Symptom scores improved at some stage for the majority of patients started on steroids for anorexia, nausea, pain, low mood, vomiting and weakness but not in patients complaining of dyspnoea or poor mobility. The most common side-effects that were most probably attributable to steroid therapy were oral candidosis and proximal myopathy. The benefits of steroids when used according to defined guidelines were thought to outweigh toxicity.

Quality of life measures for the palliative care of people severely affected by multiple sclerosis: a systematic review.

Although there is increasing interest in measuring the quality of life (QoL) of people with multiple sclerosis (MS), relatively little is known about the issues of importance to people severely affected by MS. In the first of two systematic reviews, we searched the literature to identify measures that have been used to assess health-related QoL in people with MS, and described their measurement properties in terms of validity, reliability, responsiveness to change, and appropriateness for QoL assessment in people severely affected by MS. In the second review, we identified care domains important to people with MS, by reviewing survey, focus group and interview studies involving people with MS and/or their caregivers. Forty-six studies evaluating 12 disease-specific and ten generic QoL measures for patients, and one disease-specific measure for caregivers, satisfied all inclusion criteria. Sixteen focus group or interview studies and 51 questionnaire-based studies evaluated domains of care important to people with MS, and seven qualitative and 11 questionnaire-based studies assessed domains of care important to their caregivers. From these studies, we identified 15 domains of care important to people with MS and 12 domains important to caregivers. QoL measures differed markedly in their coverage of these care domains. Moreover, each measure fulfilled some but not all criteria of validity, reliability, responsiveness, and appropriateness. Further work is needed to clarify the domains of care relevant to people with severe MS, and to measure health-related QoL in this population.

Palliative care for people severely affected by multiple sclerosis: evaluation of a novel palliative care service

Multiple sclerosis results in both physical and psychological disability but some patients have needs that are not adequately met by existing services. Our objective was to explore whether a new palliative care service improved outcomes for people severely affected by multiple sclerosis. A delayed intervention randomized controlled trial was undertaken with multiple sclerosis patients deemed by staff to have palliative care needs. The intervention was a multiprofessional palliative care team assessment and follow-up. The intervention group was offered the team immediately (fast track, FI); the control group continued best standard care and then offered the team after 3 months (standard intervention, SI). The main outcome measures were: patient reported issues using the Palliative Care Outcome Scale and Multiple Sclerosis Impact Scale at 12 weeks and caregiver burden using the Zarit Burden Inventory. Sixty-nine people were referred to the service; 52 consented or were eligible to be randomized (26 to the FI and 26 to the SI groups). At 12 weeks people in the FI group had an improvement (mean change —1.0) in the total score of 5 key symptoms whereas there was deterioration in the SI group (mean change 1.1, F = 4.75, p = 0.035). There was no difference in the change in general Palliative Care Outcome Scale or Multiple Sclerosis Impact Scale scores. There was an improvement in caregiver burden in the FI group and a deterioration in the SI group (F = 7.60, p = 0.013). Involvement with the palliative care service appeared to positively affect some key symptoms and reduced informal caregiver burden.

Loss and change: experiences of people severely affected by multiple sclerosis

This study aimed to explore important issues for people severely affected by multiple sclerosis (MS). Individual interviews were conducted with 23 people with MS (PwMS) and 17 informal carers, the data relating to 32 PwMS. Information was obtained about 19 females and 13 males, with a mean age of 55, median time from diagnosis was 14.5 years, and physical disabilities ranged from mild to severe, although fifteen patients had severe disabilities. Twenty-six of the 32 individuals were unable to walk, 24 were catheterised, and 18 had considerably impaired or no upper limb function. Personal issues in relation to loss and change, particularly in terms of losses of or changes in physical abilities, including maintaining mobility, independence, relationships and social role were raised commonly in response to an open-ended question about what issues were important in living with MS. Coping with MS requires individuals to deal with the losses and changes brought about by their illness. Our study suggests that even patients who have had MS for many years and are now severely affected continue to experience loss and change. We recommend that attention be given to emotional support which specifically addresses three main areas of dealing with loss and change for people that are severely affected - physical issues, independence and relationships. Palliative care providers may have expertise in managing loss that could be useful for these patients in partnership with neurological services.

Study protocol: delayed intervention randomised controlled trial within the Medical Research Council (MRC) Framework to assess the effectiveness of a new palliative care service

BackgroundPalliative care has been proposed to help meet the needs of patients who suffer progressive non-cancer conditions but there have been few evaluations of service development initiatives. We report here a novel protocol for the evaluation of a new palliative care service in this context.Methods/DesignUsing the MRC Framework for the Evaluation of Complex Interventions we modelled a new palliative care and neurology service for patients severely affected by Multiple Sclerosis (MS). We conducted qualitative interviews with patients, families and staff, plus a literature review to model and pilot the service. Then we designed a delayed intervention randomised controlled trial to test its effectiveness as part of phase II of the MRC framework. Inclusion criteria for the trial were patients identified by referring clinicians as having unresolved symptoms or psychological concerns. Referrers were advised to use a score of greater than 8 on the Expanded Disability Scale was a benchmark. Consenting patients newly referred to the new service were randomised to either receive the palliative care service immediately (fast-track) or after a 12-week wait (standard best practice). Face to face interviews were conducted at baseline (before intervention), and at 4–6, 10–12 (before intervention for the standard-practice group), 16–18 and 22–24 weeks with patients and their carers using standard questionnaires to assess symptoms, palliative care outcomes, function, service use and open comments. Ethics committee approval was granted separately for the qualitative phase and then for the trial.DiscussionWe publish the protocol trial here, to allow methods to be reviewed in advance of publication of the results. The MRC Framework for the Evaluation of Complex Interventions was helpful in both the design of the service, methods for evaluation in convincing staff and the ethics committee to accept the trial. The research will provide valuable information on the effects of palliative care among non-cancer patients and a method to evaluate palliative care in this context.

Randomised controlled trial of a new palliative care service: Compliance, recruitment and completeness of follow-up

BackgroundPalliative care has been proposed for progressive non-cancer conditions but there have been few evaluations of service developments. We analysed recruitment, compliance and follow-up data of a fast track (or wait list control) randomised controlled trial of a new palliative care service – a design not previously used to assess palliative care.Methods/DesignAn innovative palliative care service (comprising a consultant in palliative medicine, a clinical nurse specialist, an administrator and a psychosocial worker) was delivered to people severely affected by multiple sclerosis (MS), and their carers, in southeast London. Our design followed the MRC Framework for the Evaluation of Complex Interventions. In phase II we conducted randomised controlled trial, of immediate referral to the service (fast-track) versus a 12-week wait (standard best practice). Main outcome measures were: compliance (the extent the trial protocol was adhered to), recruitment (target 50 patients), attrition and missing data rates; trial outcomes were Palliative Care Outcome Scale and MS Impact Scale.Results69 patients were referred, 52 entered the trial (26 randomised to each arm), 5 refused consent and 12 were excluded from the trial for other reasons, usually illness or urgent needs, achieving our target numbers. 25/26 fast track and 21/26 standard best practice patients completed the trial, resulting in 217/225 (96%) of possible interviews completed, 87% of which took place in the patients home. Main reasons for failure to interview and/or attrition were death or illness. There were three deaths in the standard best practice group and one in the fast-track group during the trial. At baseline there were no differences between groups. Missing data for individual questionnaire items were small (median 0, mean 1–5 items out of 56+ items per interview), not associated with any patient or carer characteristics or with individual questionnaires, but were associated with interviewer.ConclusionThis is the first time a fast track (or wait list) randomised trial has been reported in palliative care. We found it achieved good recruitment and is a feasible method to evaluate palliative care services when patients are expected to live longer than 3–6 months. Home interviews are needed for a trial of this kind; interviewers need careful recruitment, training and supervision; and there should be careful separation from the clinical service of the control patients to prevent accidental contamination.Trial RegistrationClinical Trials.Gov NCT00364963

Symptom prevalence, severity and palliative care needs assessment using the Palliative Outcome Scale: a cross-sectional study of patients with Parkinson's disease and related neurological conditions.

Background: Palliative care is rarely being offered to patients with Parkinson’s disease. Aim: To assess symptom prevalence, severity and palliative care needs in advanced stages of Parkinsonism. Design: A cross-sectional survey using a palliative care assessment tool, the Palliative Outcome Scale was administered to patients. Setting/participants: Eight-two patients with a diagnosis of idiopathic Parkinson’s disease, multiple systems atrophy or progressive supranuclear palsy were included in the study. Results: Their mean age and disease stages 3–5 Hoehn and Yahr were 67 years and 4.1, respectively. Patients reported a mean of 10.7 (standard deviation = 3.9) physical symptoms. Over 80% had pain, fatigue, day time somnolence and problems with mobility. Other symptoms in 50%–80% included constipation, loss of bladder control, swallowing difficulties, drooling, breathlessness and sleep problems. Symptoms rated as causing severe problems were pain, fatigue, constipation and drooling. Assessment of mood revealed 70% of the patients felt anxiety and 60% had felt depressed. Eight-five per cent felt their families were anxious or worried about them. Thirty-eight per cent would have liked more information and 42% had practical problems that still needed to be addressed. There was a positive correlation between number of symptoms and disease severity (r = 0.39, p = 0.01). The total mean Palliative Outcome Scale score was 13.6 (standard deviation = 6.1), suggesting moderate palliative care needs. Conclusion: This is the first study to describe the care needs of people with Parkinson’s disease using the Palliative Outcome Scale tool. The burden of symptoms and concerns was high in advanced stages of disease. It might be appropriate that people severely affected by these conditions should be considered for referral to specialist palliative care services.

Evaluation of a new model of short-term palliative care for people severely affected with multiple sclerosis: a randomised fast-track trial to test timing of referral and how long the effect is maintained

Aims In this randomised fast-track phase II trial, the authors examined (1) whether the timing of referral to short-term palliative care (PC) affected selected outcomes, and (2) the potential staff-modifying effect of the short-term PC intervention (whether the effects were sustained over time after PC was withdrawn). Methods PC comprised a multiprofessional PC team that provided, on average, three visits, with all care completed by 6 weeks. Recruitment commenced in August 2004 and continued for 1 year. Follow-up was performed for 6 months in both groups. Outcomes were a composite measure of five key symptoms (pain, nausea, vomiting, mouth problems and sleeping difficulty) using the Palliative care Outcome Scale–MS Symptom Scale, and care giver burden was measured using the Zarit (Care Giver) Burden Interview (ZBI). Results 52 patients severely affected by multiple sclerosis were randomised to receive PC either immediately (fast-track group) or after 12 weeks (control group). Patients had a high level of disability (mean Expanded Disability Status Scale: 7.7; median: 8; SD: 1). Following PC, there was an improvement in Palliative care Outcome Scale–MS Symptom Scale score and ZBI score. A higher rate of improvement in ZBI score was seen in the fast-track group. After withdrawal of PC, effects were maintained at 12 weeks, but not at 24 weeks. Conclusions Receiving PC earlier has a similar effect on reducing symptoms but greater effects on reducing care giver burden, compared to later referral. In this phase II trial, the authors lacked the power to detect small differences. The effect of PC is maintained for 6 weeks after withdrawal but then appears to wane. Trial Registration Number National Institutes of Health, USA, http://www.Clinicaltrials.gov, NCT00364936.