Clinical Research

MPN-RC 106 Summary

The MPN-RC Research Tissue Bank plans to collect and store tissue specimens from patients with MPNs including (polycythemia vera [PV], essential thrombocythemia [ET], primary myelofibrosis [PMF]) and pre-fibrotic myelofibrosis [Pre-PMF]). Pre-PMF is a recently appreciated early indolent form of myelofibrosis (MF) which can progress to more overt forms of MF as well as to acute leukemia.   These tissue samples will be used to conduct laboratory investigations within the laboratory-based scientific projects of the MPN-RC to help define mechanisms involved in the pathophysiology, progression and effects of treatment of myelofibrosis. We anticipate enrolling 375 subjects to this study and will perform serial yearly tissue banking from each enrolled subject over a 5 year period.

Patients with the myeloproliferative neoplasms (MPNs) polycythemia vera (PV), essential thrombocythemia (ET), and primary myelofibrosis (PMF) suffer progressive cytopenias, bone marrow fibrosis and/or transformation to acute leukemia. These neoplasms are relatively rare, and therefore individual investigators at a single institution cannot obtain tissues from sufficiently large numbers of patients to reach statistically valid conclusions within a reasonable time frame.  This study is aimed to credential novel therapeutic approaches which can then be transitioned to the clinic for near-term mechanism based clinical trials in this research consortium. There is a need for new treatments for myelofibrosis (MF) patients based on laboratory insight into disease pathogenesis. We will use primary patient samples to understand how different genetic mutations contribute to MF development and to test novel treatment approaches.