Use of Thrombopoietin Receptor Agonists in Childhood Immune Thrombocytopenia

Frontiers in Pediatrics
Angelica Maria Garzon, William B Mitchell

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.

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Citations

Nov 4, 2016·Frontiers in Pediatrics·Rita ConsoliniMaria Costanza Caparello
Jul 22, 2016·Clinical Medicine Insights. Blood Disorders·Shosaku Nomura
Mar 10, 2017·Annals of Hematology·Francesco Rodeghiero, Giuseppe Carli
Sep 6, 2017·Frontiers in Pediatrics·Rita ConsoliniDavide Spatafora
Oct 30, 2018·Indian Journal of Hematology & Blood Transfusion : an Official Journal of Indian Society of Hematology and Blood Transfusion·Göksel LeblebisatanAyse Ozkan

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Clinical Trials Mentioned

NCT02201290
NCT01880047
NCT01957176
NCT01971684
NCT01071954
NCT02279173
NCT01438840
NCT00621894
NCT02139501

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