BONE MARROW TRANSPLANTATION, A POSSIBLE THERAPY FOR IDIOPATHIC THROMBOCYTOPENIC PURPURA

Abstract

Idiopathic Thrombocytopenic Purpura (ITP) is an autoimmune disease which causes the destruction of platelets. Patients who suffer from ITP can have excessive bleeding due to a lack of platelet coagulation. Current therapies to treat ITP are splenectomies and steroid treatments cause many complications and do not guarantee lasting remission. The purpose of this study is to find a more effective and permanent therapy such as bone marrow transplantation (BMT) to treat ITP using a novel mouse model. BMT can be a risky procedure, so we aim to improve it in mice by increasing the hematopoietic stem cell (HSC) homing efficiency to the bone marrow by upregulating the important chemotactic receptor, CXCR4, using compounds such as fluticasone propionate (Flonase). We found that glucocorticoids such as Flonase significantly increases CXCR4 on cultured murine HSCs, and our preliminary data suggests that HSCs double their bone marrow engraftment when treated with Flonase in competitive transplantations in wild type mice. We also found that Flonase increased chemotaxis to the CXCR4 ligand, stromal derived factor-1 in a transwell system. Second, we are creating an inducible ITP mouse model using CRISPR technology to test BMT as a possible therapy for ITP. This study can shed light on the application of BMT in ITP and a novel mouse model that can be used to test possible therapies that can have more lasting and less damaging effects than existing treatments for ITP.