This study investigates Eltrombopag as an alternative treatment pathway, which depend on stimulation of thrombocyte synthesis, in chronic and persistent immune thrombocytopenia that may be a more promising treatment than the classic type, not only by increasing platelets count but also through enhancing of the platelets activation and up regulation of GPVI expression on platelet surface.
This non-randomized single center study conducted through Ain Shams University in Egypt seeks to explore the efficacy, safety, and tolerability of eltrombopag in pediatric patients between the ages of 1-18 years who have persistent or chronic ITP. The study also seeks to better understand how eltrombopag effects platelet activation by up-regulating the glycoprotein VI (GPVI) receptor and altering it’s natural expression, in comparison to other available ITP treatments. Participants can expect to be followed every two weeks for the duration of the study and will have a baseline bone marrow examination (with reticulin stain) performed at the start as well as at the 24 week mark. Baseline liver and kidney function tests will be performed and repeated at 24 weeks into the study. Participants will also be asked to report on their quality of life using the Kid’s ITP Tools (KIT) questionnaires and this will be repeated at the end of the study.