Eltrombopag for use in children with immune thrombocytopenia

Abstract

Eltrombopag is currently the only US Food and Drug Administration–approved thrombopoietin receptor agonist for the treatment of chronic immune thrombocytopenia (ITP) in children. This oral, once-per-day therapy has shown favorable efficacy and adverse effect profiles in children. Two multicenter, double-blind, placebo controlled clinical trials (PETIT [Efficacy and Safety Study of Eltrombopag in Pediatric Patients With Thrombocytopenia From Chronic Idiopathic Thrombocytopenic Purpura (ITP)] and PETIT2 [Study of a New Medication for Childhood Chronic Immune Thrombocytopenia (ITP), a Blood Disorder of Low Platelet Counts That Can Lead to Bruising Easily, Bleeding Gums, and/or Bleeding Inside the Body]) demonstrated efficacy in raising platelet counts, reducing bleeding, and reducing the need for concomitant ITP therapies with relatively few adverse effects. The most commonly reported drug-related adverse effects include headache, nausea, and hepatobiliary laboratory abnormalities. Long-term safety data in children are limited, and studies in adults have not revealed a clinically significant increased incidence of thrombosis, marrow fibrosis, or cataract formation. Eltrombopag has also been approved for treating refractory severe aplastic anemia (AA) and has potential for expanded use in ITP and severe AA as well as in other conditions associated with thrombocytopenia.

Use of Eltrombopag in Children With Chronic Immune Thrombocytopenia (ITP): A Real Life Retrospective Multicenter Experience of the Italian Association of Pediatric Hematology and Oncology (AIEOP)

“…Moreover, the aim of eltrombopag therapy should not be to normalize platelet count. However, there are no indications regarding the platelet count recommended to start treatment (31,32). Data on eltrombopag use in children are limited to single center experiences or studies conducted outside EMA approved indications (28)(29)(30).…”

Romiplostim: Successful treatment of a pregnant woman with refractory immune thrombocytopenia

“…Also, recombinant human thrombopoietin (rhTPO) is a glycosylated form of TPO that expressed in Chinese hamster ovary cells. The rhTPO, as first-line therapy for refractory ITP, was approved by the China State Food and Drug Administration [25]. In the latest study, rhTPO was used for ITP during pregnancy.…”

Immune Thrombocytopenia in an Adolescent with Hashimoto’s Thyroiditis – Case Report

“…11,12 Several new treatment protocols have been suggested, such as eltrombopag (an oral nonpeptide thrombopoietin receptor agonist) and romiplostim (subcutaneously administered peptide mimetic thrombopoietin receptor agonist), which were found effective in children with chronic forms. 10,13 Regarding the adverse effects and safety of eltrombopag and romiplostim, these options seem to be superior to other first-line or second-line therapies.…”