Pediatric data on the use of thrombopoietin receptor agonists are fairly limited. The recent approval of eltrombopag by the US Food and Drug Administration for children aged ≥1 year, based on data from two randomized, placebo-controlled clinical trials, may lead to the increased use of this drug in clinical practice, and therefore, it is important to have a basic understanding of the biology, p...

The inefficiency of gene transfer has greatly hindered gene therapy. In vivo selection may increase the frequency of genetically modified cells, thereby circumventing this critical limitation. Here we demonstrate regulated in vivo selection in a large animal. CD34(+) cells from 2 dogs were engineered to express a conditional derivative of the thrombopoietin receptor (F36Vmpl). Activation of the...

FLT3 ligand is a hematopoietic growth factor that plays a key role in growth of primitive hematopoietic cells. FLT3 receptor mRNA is found in early hematopoietic progenitors and in human myeloid leukemia blasts. Much less is known about the surface expression of FLT3 receptor on human hematopoietic cells. Using human 125I-FLT3 ligand, we have identified and characterized surface FLT3 receptors ...

Conditioning regimens prior to hematopoietic stem cell transplantation (HSCT) are often accompanied by a period of aplasia characterized severe neutropenia, anemia, and thrombocytopenia. Long-term antibacterial immunosuppressive therapy in patients with graft-versus-host disease (GVHD) exacerbates depression. Colony-stimulating factors, erythropoietins, thrombopoietin receptor agonists used cor...

Identification of the regulatory inputs that direct megakaryocytopoiesis and platelet production is essential for the development of novel therapeutic strategies for the treatment of thrombosis and related hematologic disorders. We have previously shown that primary human megakaryocytes express the N-methyl-d-aspartate acid (NMDA) receptor 1 (NR1) subunit of NMDA-type glutamate receptors, which...

BACKGROUND Newly formed platelets are associated with increased aggregation and adverse outcomes in patients with coronary artery disease (CAD). The mechanisms involved in the regulation of platelet turnover in patients with CAD are largely unknown. AIM To investigate associations between platelet turnover parameters, thrombopoietin and markers of low-grade inflammation in patients with stabl...

Up to 30% immune thrombocytopenia (ITP) patients achieve a sustained remission off-treatment (SROT) after discontinuation of thrombopoietin receptor agonists (TPO-RAs). Factors predictive response are lacking. Patients aged ?18 years with newly diagnosed or persistent ITP were treated eltrombopag for 24 weeks. Primary end-point was SROT: the proportion responders that able taper and discontinue...

Good's syndrome is a rare acquired immunodeficiency associated with thymoma. Eltrombopag is a thrombopoietin receptor agonist and has been shown to be a valuable supplement to the treatment of several types of refractory cytopenias. In this paper, we describe a male patient suffering from Good's syndrome with immune-mediated T-cell driven pancytopenia and absence of megakaryopoiesis. He was suc...