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Cost-effectiveness and quality-of-life assessment of GM-CSF as an adjunct to intensive remission induction chemotherapy in elderly patients with acute myeloid leukaemia |
Uyl-de Groot C A, Lowenberg B, Vellenga E, Suciu S, Willemze R, Rutten F F |
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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 GM-CSF in the treatment of elderly patients with acute myeloid leukaemia (AML).
Economic study type Cost-effectiveness analysis; cost-utility analysis.
Study population Patients aged 60 or over with untreated newly diagnosed AML.
Setting The practice setting was a hospital. The economic study was carried out at the Institute for Medical Technology Assessment, Rotterdam, Holland.
Dates to which data relate Effectiveness data were collected between 1990 and 1994. It was not clear when resource data were obtained. No prices were stated.
Source of effectiveness data The evidence provided for the assessment of the cost-effectiveness of GM-CSF was obtained from a single study.
Link between effectiveness and cost data Costing was undertaken on the effectiveness study sample.
Study sample Patients admitted for AML treatment between November 1990 and October 1994 were entered into the phase 3 study and randomized to either the control group (n = 161) or the GM-CSF group (n = 157). No power calculations were reported. The control group characteristics (GM-CSF group statistics are in parentheses) were as follows: male = 52% (56%); 60-70 years = 64% (58%); 70-80 years = 33% (39%); older than 80 years = 3% (3%). Both groups had similar white blood cell counts as well as FAB cytology statistics. Eight registered patients were excluded due to incomplete data, poor physical condition, and other malignant disease.
Study design Randomised controlled trial. Subjects were randomised to either the control group (daunomycin-cytosine arabinoside without GM-CSF) or to the treatment group (daunomycin-cytosine arabinoside with GM-CSF).
Analysis of effectiveness The basis for the analysis of the clinical study was not clear. Primary health outcomes were overall and disease-free survival.
Effectiveness results Control groupsurvival statistics (GM-CSF group statistics are in parentheses) were as follows: 6 months survival = 61% (67%); 1 year = 40% (46%); 2 years = 22% (22%); 2-year disease-free survival = 19% (14%).(Note: P=0.55).
Clinical conclusions No significant differences were found in the effectiveness results between the intervention and the comparator.
Measure of benefits used in the economic analysis QALYs were the measure of benefit used in the economic analysis. These were derived using several patient questionnaires such as the Karnofsky index, the Nottingham Health Profile, and the Rotterdam Symptom Checklist. The descriptive and valuation part of the EuroQol was added to the questionnaires. Questionnaires were completed by study subjects at the start of their first treatment, during and after hospitalisation, and during follow-up at 6, 12, and 24 months.
Direct costs The price year was not stated. Discounting was performed but no rate was specified. Quantities and costs were not analysed separately. Direct costs included hospitalisation, consultations, transfusions, medication, laboratory expenses and medical procedures from the perspective of the health service.
Statistical analysis of costs Costs were analysed using theWilcoxon test.
Currency The Dutch study reported costs in US dollars ($), giving a conversion rate of $1 = 1.8 Dutch Guilders (Dfl).
Estimated benefits used in the economic analysis At induction treatment, no significant differences between the two groups were found. During hospitalisation, patients in the GM-CSF group reported more frequent problems with Karnovsky, EuroQol patient and EuroQol population scores of 63, 53.5 and 62.8 respectively (compared with scores of 75.5, 67.1 and 72.1 in the control group). The Nottingham Health Profile showed mobility and lack of energy as the major source of problems for both groups (although a lack of energy was mainly experienced by the GM-CSF group). After hospitalisation (i.e. at home), the Karnofsky scores of 84.3 (control) and 72.5 (GM-CSF) showed that subjects experienced fewer restrictions. The EuroQol patient and population scores, after hospitalisation, were 72.7 and 79.1 (control), and 68 and 66.3 (GM-CSF). At 6 months, the scores were almost equivalent (i.e. between 80.6 - 90) for both groups. At 1 year, Karnovsky and EuroQol scores were almost equivalent, although GM-CSF subjects scored better on energy and worse on pain and sleep. At 2 years, the average quality-of-life scores in patients in complete remission (CR) corresponded to the results at both 6 months and 1 year (i.e. scores were significantly better than those for no CR over the same period). However no significant differences could be proved because of the small sample sizes at this stage of the analysis (n=4 for both groups). Disease-free survival data were used to compute the Quality-adjusted Life Year (QALY) figures. The cumulative, discounted survival was 0.979 life years in the control group and 1.007 life-years in the GM-CSF group. Quality-adjusted survival (discounted) was 0.800 and 0.816 life-years in the control and GM-CSF groups, respectively.
Cost results The total (discounted) intervention cost for the control group was $51,554 compared with $57,799 for theGM-CSF group.
Synthesis of costs and benefits A cost-per-QALY analysis was not performed. Cost-effectiveness was derived from the separate analysis of costs and benefits.
Authors' conclusions Daunomycin-cytosine arabinoside combined with GM-CSF treatment is not cost-effective when compared with daunomycin-cytosine arabinoside alone.
CRD COMMENTARY - Selection of comparators The selection of the treatmentstrategies was justified.
Validity of estimate of measure of benefit The quality-of-lifeestimates of benefit used in the economic analysis are varied, in-depth and likely to be internally valid.
Validity of estimate of costs Resource data were detailed although not explicit with regard to sourcing (dates, references, etc.).
Other issues Both the costs and benefits should have been synthesised in order to arrive at a valid cost-effective or cost-ineffective conclusion. This, along with detailed sensitivity analysis would have given the conclusions more weight in deeming that daunomycin-cytosine arabinoside combined with GM-CSF treatment is not cost-effective when compared with daunomycin-cytosine arabinoside alone. A well presented and factual study.
Source of funding Supported by a grant from the Netherlands Health Council (Amstelveen, The Netherlands).
Bibliographic details Uyl-de Groot C A, Lowenberg B, Vellenga E, Suciu S, Willemze R, Rutten F F. Cost-effectiveness and quality-of-life assessment of GM-CSF as an adjunct to intensive remission induction chemotherapy in elderly patients with acute myeloid leukaemia. British Journal of Haematology 1998; 100(4): 629-636 Indexing Status Subject indexing assigned by NLM MeSH Acute Disease; Aged; Aged, 80 and over; Antineoplastic Combined Chemotherapy Protocols /economics /therapeutic use; Cost-Benefit Analysis; Cytarabine /administration & Daunorubicin /administration & Drug Costs; Female; Follow-Up Studies; Granulocyte-Macrophage Colony-Stimulating Factor /economics /therapeutic use; Hospital Costs; Humans; Karnofsky Performance Status; Length of Stay; Leukemia, Myeloid /economics /therapy; Male; Middle Aged; Prospective Studies; Quality of Life; Remission Induction; Survival Analysis; Treatment Outcome; dosage; dosage AccessionNumber 21998000523 Date bibliographic record published 31/01/1999 Date abstract record published 31/01/1999 |
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