Romiplostim in children with newly diagnosed or persistent primary immune thrombocytopenia

Immune thrombocytopenia (ITP) is a disease of heterogenous origin characterized by low platelet counts and an increased bleeding tendency. Three disease phases have been described: newly diagnosed (≤ 3 months after diagnosis), persistent (> 3–12 months after diagnosis), and chronic (> 12 month...

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Bibliographic Details
Published in:Annals of hematology 2021-09, Vol.100 (9), p.2143-2154
Main Authors: Grainger, John D., Kühne, Thomas, Hippenmeyer, Jane, Cooper, Nichola
Format: Article
Language:English
Subjects:
Child
Clinical Trials as Topic
Disease Management
Hematology
Hemorrhage - diagnosis
Hemorrhage - drug therapy
Humans
Immunotherapy
Medicine
Medicine & Public Health
Monoclonal antibodies
Oncology
Purpura, Thrombocytopenic, Idiopathic - diagnosis
Purpura, Thrombocytopenic, Idiopathic - drug therapy
Receptors, Fc - therapeutic use
Receptors, Thrombopoietin - agonists
Recombinant Fusion Proteins - therapeutic use
Review
Review Article
Thrombocytopenia
Thrombopoietin - therapeutic use
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Summary:Immune thrombocytopenia (ITP) is a disease of heterogenous origin characterized by low platelet counts and an increased bleeding tendency. Three disease phases have been described: newly diagnosed (≤ 3 months after diagnosis), persistent (> 3–12 months after diagnosis), and chronic (> 12 months after diagnosis). The majority of children with ITP have short-lived disease and will not need treatment. For children with newly diagnosed ITP, who have increased bleeding symptoms, short courses of steroids are recommended. In children who do not respond to first-line treatment or who become steroid dependent, thrombopoietin receptor agonists (TPO-RAs) are recommended because of their efficacy and safety profiles. In this narrative review, we evaluate the available evidence on the use of the TPO-RA romiplostim to treat children with newly diagnosed or persistent ITP and identify data from five clinical trials, five real-world studies, and a case report. While the data are more limited for children with newly diagnosed ITP than for persistent ITP, the collective body of evidence suggests that romiplostim is efficacious in increasing platelet counts in children with newly diagnosed or persistent ITP and may result in long-lasting treatment-free responses in some patients. Furthermore, romiplostim was found to be well tolerated in the identified studies. Collectively, the data suggest that earlier treatment with romiplostim may help children to avoid the side effects associated with corticosteroid use and reduce the need for subsequent treatment.
ISSN:0939-5555
1432-0584
DOI:10.1007/s00277-021-04590-0