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An economic evaluation of the use of granulocyte colony-stimulating factor after bone marrow transplantation in children |
Duncan N, Hewetson M, Atra A, Dick G, Pinkerton R |
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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 use of granulocyte colony stimulating factor (G-CSF), (either filgrastim or lenograstim) to reduce the severity and level of duration of neutropenia in children who have recently undergone bone marrow transplantation.
Type of intervention Treatment and secondary prevention.
Economic study type Cost effectiveness analysis.
Study population Children who had undergone autologous or allogenic bone marrow transplantation.
Setting The setting was the hospital. The economic analysis was conducted in Sutton, Surrey, United Kingdom.
Dates to which data relate Effectiveness and resource data were collected between January 1992 and January 1995. The price year was 1995.
Source of effectiveness data The evidence/estimate for final outcomes was derived from a single study.
Link between effectiveness and cost data Costs were determined retrospectively using the same population sample as in the effectiveness analysis.
Study sample 22 consecutive patients underwent either autologous (14) or allogenic (8) bone marrow transplantation and also received G-CSF therapy. There were 18 consecutive patients in the control group who did not receive G-CSF therapy following either autologous (8) or allogenic (10) bone marrow transplantation. These patients would have received G-CSF therapy had it been available at the study institution when they were treated. The median age of patients in the G-CSF group was 8.5 years and in the non G-CSF group was 5.5 years. There were 7 (32%) males in the G-CSF group compared with 15 (83%) in the non-G-CSF group. Power calculations were not used to determine the sample size.
Study design The study design was a single-centred, non-randomised trial with historic controls. The duration of follow-up was to the end of post transplant hospitalisation. There was no loss to follow-up.
Analysis of effectiveness The analysis of effectiveness was based on intention to treat. The primary health outcome was successful engraftment and discharge. A secondary health outcome measure was the absolute neutrophil recovery rate in patients.
Effectiveness results All patients had successful grafts and were discharged from hospital. Neutrophil recovery rates were significantly faster in the G-CSF group.
Clinical conclusions The effectiveness of bone marrow transplantation with or without G-CSF treatment is equally effective.
Measure of benefits used in the economic analysis Since the effectiveness analysis demonstrated that there was no significant difference in outcomes between the two groups, the economic analysis considered costs only.
Direct costs Costs were determined from the perspective of the hospital. All hospital costs that might have been affected by the use of G-CSF therapy were estimated. Transplantation costs were not included as these were common to both groups. The quantities of resources used and the costs were estimated separately. The duration of hospital stay, use of intravenous anti-microbial therapy, duration of total parenteral nutrition, duration of diamorphine (heroin) therapy, and blood/platelet use were estimated. The costs of hospitalisation, drug treatment, growth factors, and blood and laboratory tests were estimated. Drug costs were taken from the hospital pharmacy computer. Other costs were obtained from the hospital's finance department. Hospitalisation costs included staff time, overheads, supplies, hotel charges, rehabilitation and catering. Discounting was not applied, which was appropriate given the short duration of the study. Resource data in the non G-CSF group were collected between January 1992 and 1994. G-CSF group data were collected between March 1994 to January 1995. 1995 prices were used.
Statistical analysis of costs A statistical analysis of costs was carried out but the type of test used was not specified.
Indirect Costs Indirect costs were not included.
Sensitivity analysis One way sensitivity analysis was conducted to test the robustness of the economic results to changes in cost variables, the cost of bed days, anti-microbial therapy and total parenteral nutrition. Hospitalisation costs were varied 20% higher and lower than the baseline estimate. Total parenteral nutrition costs were doubled, and British National Formulary Prices were used for anti-microbials.
Estimated benefits used in the economic analysis Cost results The mean costs of treatment per patient were 15,001 (+/- 6,057, standard deviation) in the G-CSF, and 15,482 (+/- 9,613) in the non G-CSF treatment groups. This difference was not statistically significant. No significant differences were found for individual resource use between the two groups. Overall, the mean duration of hospitalisation was similar: 25.3 (+/- 4.9) days for the G-CSF, and 29.8 (+/- 16.8) days for the non G-CSF groups. G-CSF patients had a significantly mean shorter time to restoration of absolute neutrophil counts 16 days (+/- 4.7) versus 28.9 days (+/- 8.2). They also tended to have a shorter duration of anti-microbial therapy. The type of G-CSF received, either lenograstim or filgrastim, did not have an impact on costs.
Synthesis of costs and benefits Authors' conclusions There were no differences in hospitalisation or costs between children who received additional G-CSF therapy after undergoing bone marrow transplantation and those who did not. However, there may be quality of life gains for children as a result of faster absolute neutrophil count recovery due to G-CSF treatment, because these children would be released earlier from strict isolation.
CRD COMMENTARY - Selection of comparators G-CSF treatment was seen as an adjunct to standard care (the comparator) following bone marrow transplantation. You as a user of the database, should consider whether this is appropriate within your own setting.
Validity of estimate of measure of benefit Benefits were estimated from a small non-randomised trial with historic controls. The study sample appears to have been representative of the study population, although the authors did not report whether there were any significant differences in clinical characteristics between the two groups. The analysis of benefits was based upon the therapeutic equivalence of treatment options, and therefore the economic analysis only considered costs. The authors, however, noted that their analysis did not consider quality of life issues associated with a reduction in the time spent in isolation as a result of the intervention, and that these issues might be considered in a future analysis.
Validity of estimate of costs All relevant categories of costs were included for the perspective adopted by the analysis. However, some costs were excluded, such as those relating to the transplantation, as these were common to both the intervention and the comparator. In addition, the authors noted that they had not considered costs associated with pharmacy staff, and equipment costs in reconstitution procedures, which, if included, would have increased the costs of G-CSF treatment. The authors noted that costs to patients and their families were not included, due to measurement difficulties, although these should be taken into account in future analyses. Costs and resources used were correctly reported separately in the paper. Resource data were also taken from the trial. Prices were taken from the trial setting and were varied in sensitivity analysis. Discounting was not considered, which was appropriate given the short duration of the study, and a base price year was reported.
Other issues Overall this was a well constructed analysis, and the results appear to be comprehensive. The authors compared the clinical results of their study with those reported elsewhere, although they do not appear to have made comparisons with other economic analyses (if these are available in the literature). The issue of generalisability to other settings was not explicitly addressed in the analysis although some of these issues were dealt with in the sensitivity analysis
Implications of the study Further studies examining the quality of life issues around use of G-CSF treatment could be undertaken.
Source of funding Funding from Amgen Ltd, Cambridge, UK.
Bibliographic details Duncan N, Hewetson M, Atra A, Dick G, Pinkerton R. An economic evaluation of the use of granulocyte colony-stimulating factor after bone marrow transplantation in children. PharmacoEconomics 1997; 11(2): 169-174 Indexing Status Subject indexing assigned by NLM MeSH Bone Marrow Transplantation /economics /physiology; Child; Child, Preschool; Female; Granulocyte Colony-Stimulating Factor /economics /therapeutic use; Humans; Male AccessionNumber 21997008094 Date bibliographic record published 30/06/2001 Date abstract record published 30/06/2001 |
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