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Jeanne Palmer, MD
Cedars-Sinai Medical Center
Ronald Paquette, MD
Moffitt Cancer Center
Rami Komrokji, MD
University of Kansas Medical Center
Abdulraheem Yacoub, MD
University of Michigan Rogel Cancer Center
Moshe Talpaz, MD
Icahn School of Medicine at Mount Sinai
John Mascarenhas (PI), MD
Memorial Sloan Kettering Cancer Center
Raajit Rampal, MD, PhD
Wake Forest University Health Sciences
Rupali Bhave, MD
Aaron Gerds, MD
University Health Network
Vikas Gupta, MD
UT Health San Antonio
Ruben Mesa (co-PI), MD
This study is Active.
Myeloproliferative Neoplasms Research Consortium, July 2019
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.