The long-term efficacy of eltrombopag in children with immune thrombocytopenia

Li- Yang,Bao-Hua Sang,Chun-Hui Yang, Zu-Gang Xiao,Chun-Lian Fang,Yu Lv,Na Li, Qing Yang, Shu-Min Chai,Xin Tian,Xian-Wen Zhang,Ti-Long Huang

Annals of Hematology(2024)

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摘要
Immune thrombocytopenia (ITP) is the most common autoimmune disorder characterized by decreased platelet counts and impaired platelet production. Eltrombopag has been demonstrated to be safe and effective for children with ITP. It is reported eltrombopag can achieve a sustained response off treatment. However, data on its overall efficacy and safety profile are scarce in children. This study aimed to investigate the long-term efficacy of eltrombopag in children with ITP. Treatment overall response (OR), complete response (CR), response (R), durable response (DR), no response (NR), treatment free remission (TFR), and relapse rate, were assessed in 103 children with ITP during eltrombopag therapy. The OR rate, CR rate, R rate, DR rate, NR rate, TFR rate, and relapse rate were 67.0
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关键词
Eltrombopag,Children,Immune thrombocytopenia,Treatment response,Long-term efficacy,Relapse
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