Effects of erythropoietin (rHuEpo) plus granulocyte colony stimulating factor (rHuG-CSF) for the treatment of myelodysplastic syndromes (MDS) on anemia, costs and quality of life: a randomized controlled trial.

In myelodysplastic syndromes (MDS), anemia responds to erythropoietin (rHuEpo) alone and in combination with granulocyte colony-stimulating factor (rHuG-CSF) in 10-20% and 35-40% of cases respectively. We randomly divided 60 low-grade anemic MDS patients with serum Epo levels <500 mU/ml into two groups: rHuEpo plus rHuG-CSF (arm A) or supportive care (arm B). After 12 weeks, erythroid responders were given rHuEpo alone for 40 additional weeks. They were also given rHuG-CSF if they relapsed. Responses were considered to be major if the hemoglobin (Hb) level (cid:1) 11.5 g/dl or minor if Hb levels were (cid:1) 1.5 g/dl or the Hb level remained stable without transfusion. There were 10/24 responders in arm A and 0/26 in arm B (p=0.01). Eight patients in arm A continued rHuEpo alone and six relapsed. The response was always restored in when rHuG-CSF was reintroduced. Mean direct costs per patient were 26723 euros (arm A) and 8746 euros (arm B). Quality of life was assessed with a FACT-An scale. Similar percentages of patients from both arms showed a significant clinical improvement. rHu-Epo plus rHuG-CSF led to a response in 41.7 % of MDS patients.This treatment was expensive. No effect on quality of life was demonstrated.

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