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Phase I/II trial of PIXY321 (granulocyte‐macrophage colony stimulating factor/interleukin‐3 fusion protein) for treatment of inherited and acquired marrow failure syndromes
Author(s) -
Taylor Douglas S.,
Lee Yisheng,
Sieff Colin A.,
Homans Alan,
Garrison Leslie,
Guinan Eva C.
Publication year - 1998
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1046/j.1365-2141.1998.01012.x
Subject(s) - medicine , bone marrow , absolute neutrophil count , granulocyte colony stimulating factor , granulocyte , toxicity , bone marrow failure , granulocyte macrophage colony stimulating factor , platelet , gastroenterology , immunology , cytokine , chemotherapy , neutropenia , haematopoiesis , stem cell , biology , genetics
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/m 2 /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.