COST-EFFECTIVENESS AND QUALITY-OF-LIFE ASSESSMENT OF GM-CSF AS AN ADJUNCT TO INTENSIVE REMISSION INDUCTION CHEMOTHERAPY IN ELDERLY PATIENTSWITH ACUTE MYELOID-LEUKEMIA

We conducted a prospective, randomized, multicentre clinical trial com paring the effects and costs of GM-CSF as an adjunct to intensive chem otherapy in elderly patients with acute myeloid leukaemia (AML). The p atients were randomized to either daunomycin-cytosine arabinoside (con trol arm: rr = 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, C R after remission induction treatment was achieved in 55% of the patie nts in the control group and in 56% of the patients in the GM-CSF grou p (P = NS), The duration of survival and disease-free survival at 2 ye ars 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 s hort-term quality of life, the administration of GM-CSF resulted in mo re 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 t reatment were higher in GM-CSF-treated patients than in the control gr oup, i.e. US$40782 and US$34465, respectively (P < 0.01). The costs du ring the follow-up period did not differ between the two groups. The r esults of this randomized clinical trial indicate that daunomycin-cyto sine arabinoside plus GM-CSF is not a cost-effective treatment strateg y when compared with daunomycin-cytosine arabinoside alone.