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Blood, 15 July 2002, Vol. 100, No. 2, pp. 728-730
BRIEF REPORT
Effects of pegylated recombinant human megakaryocyte growth and
development factor in patients with idiopathic
thrombocytopenic purpura
Shosaku Nomura,
Kazuo Dan,
Tomomitsu Hotta,
Kingo Fujimura, and
Yasuo Ikeda
From the First Department of Internal Medicine, Kansai
Medical University, Osaka, Japan; the Division of Hematology,
Department of Internal Medicine, Nippon Medical School; the Department
of Hematology and Rheumatology, Tokai University School of Medicine,
Kanagawa, Japan; and the Department of Clinical Pharmaceutical Science,
Graduate School of Medicine, Hiroshima University, Hiroshima, Japan;
and the Division of Hematology, Department of Internal Medicine, Keio
University School of Medicine, Tokyo, Japan.
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 × 109/L. Four patients received PEG-rHuMGDF
(0.5 µg/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 × 109/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.
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