Effects of pegylated recombinant human megakaryocyte growth and development factor in patients with idiopathic thrombocytopenic purpura.
We conducted a phase 1-2 clinical trial to evaluate the effect of pegylated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF) in patients with chronic idiopathic thrombocytopenic purpura (ITP) refractory to standard therapy who had platelet counts below 30 x 10(9)/L. Four patients received PEG-rHuMGDF (0.5 microg/kg of body weight per day) by daily intravenous injection for up to 7 days. Administration of PEG-rHuMGDF increased platelet counts in 3 patients. A striking thrombocytosis occurred in 2 patients, whose platelet counts were elevated to more than 700 x 10(9)/L a week after the last administration of PEG-rHuMGDF and returned to baseline levels within 4 to 6 weeks. Before the platelet peak, the percentage of reticulated platelets increased transiently in 3 patients tested, including one patient who had no response. Bleeding episodes decreased after the start of PEG-rHuMGDF therapy. These results suggest that PEG-rHuMGDF might have a clinical benefit in ameliorating thrombocytopenia associated with ITP.
Nomura
Shosaku
S
First Department of Internal Medicine, Kansai Medical University, Osaka, Japan.
Dan
Kazuo
K
Hotta
Tomomitsu
T
Fujimura
Kingo
K
Ikeda
Yasuo
Y
D000328:Adult; D000368:Aged; D015331:Cohort Studies; D005260:Female; D006470:Hemorrhage / Q000188:drug therapy; D006801:Humans; D008297:Male; D008875:Middle Aged; D010976:Platelet Count; D011092:Polyethylene Glycols / Q000008:administration & dosage* / Q000494:pharmacology; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000150:complications / Q000188:drug therapy*; D011994:Recombinant Proteins / Q000008:administration & dosage* / Q000494:pharmacology; D016879:Salvage Therapy; D013922:Thrombocytosis / Q000139:chemically induced; D013926:Thrombopoietin / Q000008:administration & dosage* / Q000494:pharmacology; D016896:Treatment Outcome
D011994:Recombinant Proteins; C103117:polyethylene glycol-recombinant human megakaryocyte growth and development factor; D011092:Polyethylene Glycols; D013926:Thrombopoietin