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

Douglas S. Taylor, Yisheng Lee, Colin A. Sieff, Alan Homans, Leslie Garrison, Eva C. Guinan

Research output: Contribution to journalArticle

8 Scopus citations

Abstract

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 750mg/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.

Original languageEnglish (US)
Pages (from-to)304-307
Number of pages4
JournalBritish Journal of Haematology
Volume103
Issue number2
DOIs
StatePublished - 1998
Externally publishedYes

Keywords

  • Bone marrow failure
  • Children
  • Growth factor
  • Lymphokine
  • PIXY321

ASJC Scopus subject areas

  • Hematology

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