Activity of STI571 in chronic myelomonocytic leukemia with a platelet-derived growth factor β receptor fusion oncogene

摘要: Platelet-derived growth factor β receptor (PDGFβR) fusion genes have been shown to be critical transforming oncogenes in a subset of patients with chronic myelomonocytic leukemia (CMML). The sensitivity dysregulated tyrosine kinase the inhibitor STI571 (imatinib mesylate) makes it potentially attractive treatment option this patients. We recently cloned novel member PDGFβR oncogene family, rabaptin-5-PDGFβR. A patient CMML carrying rabaptin-5-PDGFβR gene underwent allogeneic stem cell transplantation (SCT) and was monitored closely sensitive reverse transcriptase–polymerase chain assay detect transcript. After achieving molecular remission at 5 months after transplantation, 15 SCT showed persistent progressive evidence relapse. demonstrating vitro that cells transformed specific are efficiently killed by STI571, started on STI571. responded rapidly entered 6 weeks therapy, he continues later. These results suggest may an effective targeted therapy related oncogenes.