title: Cost-effectiveness and quality-of-life assessment of GM-CSF as an adjunct to intensive remission induction chemotherapy in elderly patients with acute myeloid leukemia.
abstract: We conducted a prospective, randomized, multicentre clinical trial comparing the effects and costs of GM-CSF as an adjunct to intensive chemotherapy in elderly patients with acute myeloid leukaemia (AML). The patients were randomized to either daunomycin-cytosine arabinoside (control arm: n = 161) or daunomycin-cytosine arabinoside with GM-CSF (GM-CSF arm: n = 157). The primary end-point was the effect of GM-CSF on the percentage of complete remissions (CR). Survival duration, disease-free survival, quality of life and costs were evaluated separately. CR after remission induction treatment was achieved in 55% of the patients in the control group and in 56% of the patients in the GM-CSF group (P = NS). The duration of survival and disease-free survival at 2 years after randomization were estimated at 22% and 19% for the control group and 22% and 14% for the GM-CSF group (P = NS). Considering the short-term quality of life, the administration of GM-CSF resulted in more problems with regard to depressed mood, diarrhoea and rash/eczema. With regard to the long-term quality of life there were no significant differences between the two groups. The average costs of the primary treatment were higher in GM-CSF-treated patients than in the control group, i.e. US$40782 and US$34465, respectively (P < 0.01). The costs during the follow-up period did not differ between the two groups. The results of this randomized clinical trial indicate that daunomycin-cytosine arabinoside plus GM-CSF is not a cost-effective treatment strategy when compared with daunomycin-cytosine arabinoside alone.
authors.affiliation: Institute for Medical Technology Assessment/Department of Health Policy and Management, Erasmus University Rotterdam, The Netherlands.
authors.forename: C A
authors.initials: CA
authors.lastname: Uyl-de Groot
authors.forename: B
authors.initials: B
authors.lastname: Löwenberg
authors.forename: E
authors.initials: E
authors.lastname: Vellenga
authors.forename: S
authors.initials: S
authors.lastname: Suciu
authors.forename: R
authors.initials: R
authors.lastname: Willemze
authors.forename: F F
authors.initials: FF
authors.lastname: Rutten
mesh_terms: D000208:Acute Disease; D000368:Aged; D000369:Aged, 80 and over; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000191:economics / Q000627:therapeutic use*; D003362:Cost-Benefit Analysis; D003561:Cytarabine / Q000008:administration & dosage; D003630:Daunorubicin / Q000008:administration & dosage; D016527:Drug Costs; D005260:Female; D005500:Follow-Up Studies; D016178:Granulocyte-Macrophage Colony-Stimulating Factor / Q000191:economics / Q000627:therapeutic use*; D017721:Hospital Costs; D006801:Humans; D017567:Karnofsky Performance Status; D007902:Length of Stay; D007951:Leukemia, Myeloid / Q000191:economics / Q000628:therapy*; D008297:Male; D008875:Middle Aged; D011446:Prospective Studies; D011788:Quality of Life; D012074:Remission Induction; D016019:Survival Analysis; D016896:Treatment Outcome
chemical_list: D003561:Cytarabine; D016178:Granulocyte-Macrophage Colony-Stimulating Factor; D003630:Daunorubicin