Use of Thrombopoietin Receptor Agonists in Childhood Immune Thrombocytopenia

Abstract

Most children with immune thrombocytopenia (ITP) will have spontaneous remission regardless of therapy, while about 20% will go on to have chronic ITP. In those children with chronic ITP who need treatment, standard therapies for acute ITP may have adverse effects that complicate their long-term use. Thus, alternative treatment options are needed for children with chronic ITP. Thrombopoietin receptor agonists (TPO-RA) have been shown to be safe and efficacious in adults with ITP, and represent a new treatment option for children with chronic ITP. One TPO-RA, eltrombopag, is now approved for children. Clinical trials in children are ongoing and data are emerging on safety and efficacy. This review will focus on the physiology of TPO-RA, their clinical use in children, as well as the long-term safety issues that need to be considered when using these agents.

Keywords: chronic immune thrombocytopenia; eltrombopag; immune thrombocytopenia; romiplostim; thrombopoietin receptor agonists.

Figure 1

Thrombopoietin receptor activation by thrombopoietin, romiplostim, and eltrombopag via the STAT/MAPK pathways for increased platelet production. This research was originally published in International Journal of Hematology [Kuter (9) by The Japanese Society of Hematology].