Use of Thrombopoietin Receptor Agonists in Childhood Immune Thrombocytopenia

Angelica Maria Garzon, William Beau Mitchell

Research output: Contribution to journalReview articlepeer-review

17 Scopus citations


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.

Original languageEnglish (US)
Article number70
JournalFrontiers in Pediatrics
StatePublished - Aug 13 2015
Externally publishedYes


  • chronic immune thrombocytopenia
  • eltrombopag
  • immune thrombocytopenia
  • romiplostim
  • thrombopoietin receptor agonists

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health


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