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Home based management in multiple sclerosis: results of a randomised controlled trial |
Pozzilli C, Brunetti M, Amicosante A M, Gasperini C, Ristori G, Palmisano L, Battaglia M |
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Record Status This is a critical abstract of an economic evaluation that meets the criteria for inclusion on NHS EED. Each abstract contains a brief summary of the methods, the results and conclusions followed by a detailed critical assessment on the reliability of the study and the conclusions drawn. Health technology The health intervention examined in the study was home-base management of patients with multiple sclerosis. Patients were followed through home visits and telephone calls by a multidisciplinary team including two neurologists, an urologist, a rehabilitation physician, a psychologist, a physical therapist, a nurse, a social worker, and a co-ordinator. A dedicated phone number was made available Monday to Friday, 9 a.m. till 5 p.m. and members of the team were provided with cell phones so that they could be easily reached for advice or to arrange a visit. The intervention consisted of observation, administration of intravenous drugs, nursing care, rehabilitation of the patients, patient and caregiver education, psychological support, and the services of the social secretariat.
Economic study type Cost-effectiveness analysis.
Study population The study population comprised patients affected by clinically definite multiple sclerosis.
Setting The setting was hospital. The economic study was conducted at the multiple sclerosis centre of the neurological department of La Sapienza University in Rome, Italy.
Dates to which data relate Data on effectiveness and resource use were gathered between January 1997 and January 1998. The price year was 1999.
Source of effectiveness data A single study was used to derive effectiveness data.
Link between effectiveness and cost data The costing was conducted prospectively on the same patient sample as that used in the effectiveness analysis.
Study sample Power calculations were performed and showed that a total sample of 201 patients (with a 2:1 randomisation ratio and assuming a 10% drop-out rate) would be required to detect statistically significant differences, in terms of the primary health outcome, with 90% power. All eligible patients, assessed for suitability by the co-ordinator of the home-based care team in the Rome service area, were included in the study. Of the 297 initially eligible, consecutive patients screened, 201 subjects agreed to participate in the study: 133 patients (mean age: 47+/-10.3 years; 65% women; disease duration: 18.4+/-9.5 years) were included in the intervention group (home-based care) and 68 patients (mean age: 46.7+/-13.3 years; 69% women; disease duration: 18.6+/-11 years) in the control group (traditional hospital care). Of the 96 patients who refused to participate, 62 patients were not sure that home management would be appropriate for their situation and 34 patients declined the invitation as they had only been referred to the multiple sclerosis centre for a second opinion, and were being regularly followed by their own neurologists.
Study design This was a randomised controlled trial, carried out in several institutions in Rome, Italy. Patients were randomised (in blocks of six) to the two study groups in a ratio of 2:1 using a computer-generated algorithm. Randomisation was stratified by age and expanded disability status score (EDSS). Patients were followed for a period of one year and at the end of the follow-up period, data were available for 123 patients in the intervention group and 65 in the control group.
Analysis of effectiveness The basis for the analysis of the clinical study (intention to treat or treatment completers only) was not reported. The primary health outcome was quality of life, estimated using the 36-item short form health survey questionnaire (SF-36). The secondary health outcomes were; assessment of neurological impairment and cognitive abilities, evaluated using the EDSS and the mini-mental state examination (MMSE); disability and fatigue, through the functional independence measure (FIM) and fatigue severity scale (FSS); and mood status, using the state trait anger expression inventory (STAXI), the state trait anxiety inventory (STAI), and the clinical depression questionnaire (CDQ). Study groups were generally comparable at baseline, but four of the eight domains used in the SF-36 were statistically lower in the intervention group, thus resulting in worse baseline quality of life. To take into account these potentially confounding factors, the authors performed a regression analysis where outcome scores were adjusted for baseline values.
Effectiveness results The changes from baseline to one-year assessment in functional outcomes in terms of EDSS, FIM, MMSE, CDQ, FSS, STAI, and STAXI were not statistically different and results were not reported.
The SF-36 scores improved in both study groups, but improvements in the intervention group were statistically more consistent than in the control group in terms of bodily pain (difference: 3.46; 95% CI: 2.38 - 4.54), general health (difference: 5.01; 95% CI: 4.50 - 5.51), social functioning (difference: 1.09; 95% CI: 0.51 - 1.67), and role, emotional (difference: 12.39; 95% CI: 9.85 - 14.93).
When the eight dimensions were combined into two scores, physical component score (PCS) and mental component score (MCS), the improvements in the intervention group were statistically higher than in the control group. For PCS the difference was 1.19 (95% CI: 1.04 - 1.34) and for MCS the difference was 0.75 (95% CI: 0.58 - 0.91).
Clinical conclusions The effectiveness analysis showed that home-based care significantly improved quality of life as perceived by patients with multiple sclerosis in comparison with standard hospital-based care. No difference was found in terms of measures of neurological impairment/disability.
Measure of benefits used in the economic analysis Although statistically significant differences were found in terms of some dimensions of quality of life between the study groups, the authors did not use a summary benefit measure and undertook a cost-minimisation analysis.
Direct costs Discounting was not conducted, nor relevant as costs were incurred over a period of one year. Unit costs and quantities of resources were not reported separately. The health service costs included in the analysis were inpatient care (ordinary care, rehabilitation, day-hospital, and diagnostic tests), outpatient care, home care, and home-based co-ordination programme. Costs of drugs and aids for daily life activities were not included in the analysis. The cost/resource boundary adopted was that of the Italian National Health Service. The inpatient costs were estimated using the Italian third party payer reimbursement for diagnosis-related groups (DRGs) as a proxy for real costs. The outpatient costs were derived from National Health Service tariffs and an additional fee was added for domiciliary visits. Quantities of resources used were estimated alongside the clinical trial in the period January 1997-January 1998. The price year was 1999.
Statistical analysis of costs Standard statistical analyses of costs were conducted to test for statistical significance of results.
Indirect Costs Indirect costs were not included in the analysis.
Currency Costs were estimated in Italian lira (L), and then converted and reported in Euros. No conversion rate was reported.
Sensitivity analysis Multivariate sensitivity analyses were conducted to estimate the robustness of the cost estimates by constructing a best case scenario (an increase of 10% in reimbursement for admissions, a decrease of 10% for home-based management costs, and the upper limit of the 95% confidence interval) and a worst case scenario (a decrease of 10% in reimbursement for admissions, an increase of 10% for home-based management costs, and the lower limit of the 95% confidence interval).
Estimated benefits used in the economic analysis Please refer to the effectiveness results above.
Cost results Total costs per patient were Euro 1,443 in the intervention group and Euro 2,265 in the control group, with cost-savings of Euro 822 per patient favouring the home-based care. This difference was mainly due to fewer inpatient days. In the sensitivity analysis, the cost-savings associated with home-based management over the traditional hospital-based care ranged from Euro 2,086 (best case scenario) to Euro 234 (worst case scenario).
Synthesis of costs and benefits Costs and benefits were not combined as a cost-minimisation analysis was conducted.
Authors' conclusions The authors concluded that the multidisciplinary approach for home-based care improved the quality of life in patients with multiple sclerosis without increasing costs.
CRD COMMENTARY - Selection of comparators The rationale for the choice of the comparator was clear. Hospital-based care was selected as it represented the standard management approach for patients with multiple sclerosis. You, as a user of this database, should decide whether it is a valid comparator in your own setting.
Validity of estimate of measure of effectiveness The analysis of effectiveness was based on a randomised controlled trial, which was appropriate for the study question. The methods of randomisation and sample selection were reported. The study sample was representative of the study population. The authors conducted some statistical analyses to take into account baseline differences in the study groups, thus limiting bias and confounding factors. Power calculations were performed. These issues tend to increase the internal validity of the analysis. The authors commented that, for obvious reasons, outcome assessment was not fully blinded and this could have had some impact on the analysis, as well as the fact that patients with a strong preference for hospital care refused to participate.
Validity of estimate of measure of benefit No summary benefit measure was used in the economic analysis. The analysis was therefore categorised as a cost-consequences study (see validity of estimate of measure of effectiveness comments above).
Validity of estimate of costs The analysis of costs was conducted from the perspective of the Italian National Health Service and it appears that all relevant categories of costs were included in the analysis. Some costs, such as pharmaceuticals and aids for daily life activities were excluded from the economic evaluation, but the authors did not comment on the impact of such exclusions. The price year was reported, thus making reflation exercises in other settings possible. Statistical analyses of quantities were conducted and sensitivity analyses were performed on unit costs. The source of cost data was reported for the different items included in the analysis. However, unit costs were not reported. The authors commented that estimated total costs differed from those estimated in other studies, due to different methods of resource data collection and the use of reimbursement rates to approximate true costs. As a result of the methods used to estimate inpatient costs the results may not be generalisable to other settings.
Other issues The authors made some comparisons of their findings with those from other studies, but did not address the issue of the generalisability of the study results to other settings. Sensitivity analyses were conducted only on the costs of the interventions. The study enrolled patients with multiple sclerosis and this was reflected in the conclusions of the analysis. The authors appear to have presented their results selectively.
Implications of the study The main implication of the analysis was that home-based care proved to be appropriate for patients with multiple sclerosis and could be used as a complement to traditional hospital care, above all for patients who live at home, but who are subject to long periods of hospitalisation. The authors noted that future research should investigate the relationship between home-based care and both hospital and community services in patients suffering from multiple sclerosis.
Source of funding Fondazione Italiana Sclerosi Multipla, the Istituto Superiore di Sanita, and Dompe Biotec.
Bibliographic details Pozzilli C, Brunetti M, Amicosante A M, Gasperini C, Ristori G, Palmisano L, Battaglia M. Home based management in multiple sclerosis: results of a randomised controlled trial. Journal of Neurology, Neurosurgery and Psychiatry 2002; 73(3): 250-255 Indexing Status Subject indexing assigned by NLM MeSH Adult; Cost-Benefit Analysis; Disability Evaluation; Female; Follow-Up Studies; Health Care Costs; Home Care Services, Hospital-Based /economics; Humans; Male; Middle Aged; Multiple Sclerosis /economics /rehabilitation; Patient Care Team; Research Support, Non-U.S. Gov't AccessionNumber 22002001579 Date bibliographic record published 31/05/2003 Date abstract record published 31/05/2003 |
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