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 ram domization 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 longterm 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 costeffective treatment strategy when compared with daunomycin-cytosine arabinoside alone.

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doi.org/10.1046/j.1365-2141.1998.00635.x, hdl.handle.net/1765/66419
British Journal of Haematology
Department of Hematology

Uyl-de Groot, C.A, Löwenberg, B, Vellenga, E, Suciu, S, Willemze, R, & Rutten, F.F.H. (1998). 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, 100(4), 629–636. doi:10.1046/j.1365-2141.1998.00635.x