Bone marrow endothelium plays an important role in the homing of hematopoietic stem and progenitor cells (HSPC) upon transplantation. Irradiation promotes vascular permeability as a consequence of temporal loss of vascular endothelial-cadherin function. This enhances homing of HSPC to the bone marrow.

This phase II dose modification study investigated the safety and efficacy of eltrombopag in 30 patients with low risk myelodysplastic syndromes. Forty-four perecent of patients responded, 20% and 24% had uni- or bi-lineage hematologic responses, respectively. Three patients developed reversible grade 3 liver toxicity and one patient had increased reticulin fibrosis.