In einer Phase-I/II-Studie wurde der therapeutische Effekt von rhGM-CSF bei Patienten mit myelodysplastischen Syndromen analysiert. 9 Patienten, für die keine anderen Therapiemöglichkeiten bestanden, erhielten rhGM-CSF in einer Dosierung von 15 μg/m2 bis 150 μg/m2 als 8stündige i.v.-Infusion für 7 Tage. Nach einem therapiefreien Intervall von 14 Tagen wurde der Therapiezyklus wiederholt. Dosisabhängig stiegen die Leukozyten bei 7 von 9 Patienten an, während die Throm-bozyten nur bei 1 Patienten anstiegen. Veränderungen der Retikulozyten wurden nicht beobachtet. rhGM-CSF wurde gut toleriert, Hauptnebenwirkungen waren leichte lokale Phlebitiden an der Infusions-stelle und Sternalschmerzen nach Bolusinjektion.

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