Clinical potential of pacritinib in the treatment of myelofibrosis

摘要: Myelofibrosis (MF) is a myeloid disorder caused by clonal hematopoietic stem-cell proliferation associated with activation of the Janus kinase (JAK) signal transducer and activator transcription (STAT) signaling pathways. Patients MF often develop severe splenomegaly, marked symptom burden significant cytopenias, consequent negative impact on quality life survival. The management patients has dramatically improved development group drugs that inhibit JAK signaling. first these agents to be approved was ruxolitinib, JAK1/JAK2 inhibitor, which been shown improve both spleen size symptoms in MF. However, myelotoxicity, particularly platelet lineage, significantly limits patient population who can benefit from this agent. Thus, there an unmet need for novel limited myelotoxicity treat Pacritinib, JAK2 FMS-like tyrosine 3 (FLT3) promising results early phase trials clinical responses are comparable those seen even thrombocytopenia. Currently two large III pacritinib MF, including thrombocytopenia, previously treated ruxolitinib. If encouraging observed confirmed, will represent new exciting treatment option cytopenias.