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 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.

Grainger JD ，Kühne T ，Hippenmeyer J ，Cooper N ... -  《-》

Romiplostim in adults with newly diagnosed or persistent immune thrombocytopenia.

Lozano ML ，Godeau B ，Grainger J ，Matzdorff A ，Rodeghiero F ，Hippenmeyer J ，Kuter DJ ... -  《-》

Effectiveness and safety of romiplostim among patients with newly diagnosed, persistent and chronic ITP in routine clinical practice in central and Eastern Europe: an analysis of the PLATON study.

Skopec B ，Sninska Z ，Tzvetkov N ，Ivanushkin V ，Björklöf K ，Hippenmeyer J ，Mihaylov G ... -  《-》

Thrombopoietin Receptor Agonists: Eltrombopag and Romiplostim for the Treatment of Chronic Immune Thrombocytopenia Purpura.

Atkinson K 《-》

Thrombopoietin Receptor Agonists in Children with Immune Thrombocytopenia: A New Therapeutic Era.

Immune thrombocytopenia (ITP) is a common bleeding disorder in childhood. The management of ITP in children is controversial, requiring personalized assessment of patients and therapeutic choices. Thrombopoietin receptor agonists (TPO-RAs), eltrombopag and romiplostim, have been shown to be safe and effective for the treatment of pediatric ITP. The aim of our research is to define the role of thrombopoietin receptor agonists in the management of pediatric ITP. This review focuses on the use of TPO-RAs in pediatric ITP, in randomized trials and in clinical routine, highlighting their key role in the management of the disease. Eltrombopag and romiplostim appear effective treatment options for children with ITP. Several clinical studies have assessed that the use of TPO-RAs increases platelet count, decreases bleeding symptoms and improves health-related quality of life. Moreover, TPO-RAs are well tolerated with minor side effects. Although long term efficacy and safety of TPO-RAs still require further investigations, their use is gradually expanding in the clinical practice of children with ITP.

Lassandro G ，Palladino V ，Vecchio GCD ，Palmieri VV ，Corallo PC ，Faienza MF ，Giordano P ... -  《-》