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Blood. 2002 Jul 15;100(2):728-30.

Effects of pegylated recombinant human megakaryocyte growth and development factor in patients with idiopathic thrombocytopenic purpura.

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  • 1First Department of Internal Medicine, Kansai Medical University, Osaka, Japan.

Abstract

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.

PMID:
12091377
[PubMed - indexed for MEDLINE]
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