Phase I/II trial of PIXY321 (granulocyte-macrophage colony stimulating factor/interleukin-3 fusion protein) for treatment of inherited and acquired marrow failure syndromes.

Autor: Taylor, Douglas S., Lee, Yisheng, Sieff, Colin A., Homans, Alan, Garrison, Leslie, Guinan, Eva C.
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Zdroj: British Journal of Haematology; Nov98, Vol. 103 Issue 2, p304-307, 4p, 2 Charts
Abstrakt: Fourteen paediatric patients with advanced amegakaryocytic thrombocytopenia (AMT) or other bone marrow (BM) failure syndromes were enrolled on one of two phase I/II dose escalation studies of PIXY321. PIXY321 was administered subcutaneously in doses ranging from 250 to 750 mg/m2/d. No dose-limiting toxicity was observed. Peak absolute neutrophil count (ANC) was higher than baseline in all patients. Most transfusion-independent patients demonstrated elevation in haematocrit and/or platelet count. Trilineage haemopoietic responsiveness was evident in the three transfusion-independent patients. In these paediatric populations PIXY321 is well tolerated and merits consideration as a potential therapy. [ABSTRACT FROM AUTHOR]
Databáze: Complementary Index
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