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Open lable phase II study of metformin activity in patients with primary myelofibrosis, post-polycythemia vera and essential post-thrombocythemia

Grant number: 19/03920-0
Support Opportunities:Research Grants - Research in Public Policies
Duration: September 01, 2019 - February 28, 2022
Field of knowledge:Health Sciences - Medicine - Medical Clinics
Convênio/Acordo: CNPq - PPSUS
Principal Investigator:Fabíola Traina
Grantee:Fabíola Traina
Host Institution: Faculdade de Medicina de Ribeirão Preto (FMRP). Universidade de São Paulo (USP). Ribeirão Preto , SP, Brazil


Myeloproliferative neoplasia originates from the hematopoietic stem cell, are characterized by JAK/STAT signaling activation, and decontrolled proliferation of one or more cells of the myeloid lineage. Patients may present changes in hematimetric counts, splenomegaly, and thromboembolic events, and evolve with increased fibrosis in the bone marrow and acute myeloid leukemia. The only potentially curative treatment is haematopoietic stem cell transplantation, which is associated with high morbidity and mortality and is reserved for young patients with a compatible donor. The selective JAK1/2 inhibitor reduces constitutional symptoms without modifying the natural history of the disease and is not available in the public health care system due to its high cost. Our research group determined the preclinical benefits of metformin in JAK2V617F cells: reduction of cell viability, inhibition of the JAK/STAT pathway and mitochondrial activity in vitro, and reduction of splenomegaly in vivo. The aim of this study will be to evaluate the efficacy, safety and tolerability of metformin in patients with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis and essential post-thrombocythemia myelofibrosis. Females and males patients, older than 18 years, who have been diagnosed with PMF, post-PV myelofibrosis and post-ET myelofibrosis will be eligible. The study will be carried out in two phases, 10 patients will be included in the first phase, and if one of the patients responds, 5 additional patients will be recruited in the second phase. The drug will be considered effective if at least 2 patients respond. Response will be defined as reduction e 35% in spleen volume from the start of treatment to week 24 as measured by computed tomography without contrast or magnetic resonance imaging of the abdomen. Exploratory assessments will include evaluation of the hemostatic balance. (AU)

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