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Effect of eltrombopag on the expansion of hematopoietic stem and progenitor cells in the treatment of aplastic anemia

Grant number: 18/25178-0
Support type:Scholarships in Brazil - Scientific Initiation
Effective date (Start): March 01, 2019
Effective date (End): February 29, 2020
Field of knowledge:Health Sciences - Medicine
Principal Investigator:Rodrigo do Tocantins Calado de Saloma Rodrigues
Grantee:Bruno Quintino de Oliveira
Home Institution: Faculdade de Medicina de Ribeirão Preto (FMRP). Universidade de São Paulo (USP). Ribeirão Preto , SP, Brazil

Abstract

Acquired aplastic anemia is a disease in which hematopoietic stem cell failure occurs, leading to pancytopenia associated with hypocellular bone marrow. The disease is caused by the destruction of hematopoietic precursors by a cytotoxic T cell mediated immune mechanism. Historically, the mortality of this disease has always been high, both due to acute complications of pancytopenia and complications inherent to its treatment. The current treatment for this disease consists of immunosuppressive therapy or related allogeneic hematopoietic stem cell transplantation. However, recent studies have shown that eltrombopag, a thrombopoietin receptor agonist, while associated with immunosuppressive therapy, significantly improves hematologic response, increasing bone marrow cellularity, the quantity of cells from the three blood lineages in peripheral blood, transfusion independence and overall survival. In addition, the percentage of cases with complete hematologic response, with blood count normalization, is significantly higher than the results of isolated immunosuppression, suggesting a direct effect on the recovery of hematopoiesis and its precursors. This study aims to evaluate the effects of eltrombopag on the expansion of hematopoietic stem cells and progenitors, determining the composition and phenotype of the cell populations that are reestablished in the bone marrow.