Efficacy and safety of eltrombopag in the treatment of Chinese children with chronic immune thrombocytopenia

• Published in: Hematology (Luxembourg) 2021-01, Vol.26 (1), p.31-36
• Main Authors: Chen, Mo, Fang, Jian-Pei, Zhou, Chuan-Xin, Li, Xin-Yu, Lin, Shao-Fen, Xu, Lu-Hong
• Format: Article
• Language: English
• Subjects: Benzoates - adverse effects; Benzoates - therapeutic use; Child; Child, Preschool; children; China - epidemiology; Chronic Disease; Chronic immune thrombocytopenia; efficacy; eltrombopag; Female; Humans; Hydrazines - adverse effects; Hydrazines - therapeutic use; Infant; Male; megakaryocyte; Purpura, Thrombocytopenic, Idiopathic - drug therapy; Purpura, Thrombocytopenic, Idiopathic - epidemiology; Pyrazoles - adverse effects; Pyrazoles - therapeutic use; Receptors, Thrombopoietin - agonists; regulatory T lymphocyte; Retrospective Studies; safety; T-Lymphocytes, Regulatory - drug effects; treatment; Treatment Outcome
• ISSN: 1607-8454; 1607-8454
• DOI: 10.1080/16078454.2020.1856511

Our aim is to evaluate initial efficacy, safety, and durable response of eltrombopag in the treatment of Chinese children with chronic immune thrombocytopenia (cITP). This was a retrospective, single-center cohort study including 30 pediatric patients with cITP administered eltrombopag between 1 July 2017 and 1 January 2019. Patients with at least 12 weeks of eltrombopag treatment and available follow-up data were included. Initial response rate, durable response rate, bleeding events, and adverse events were assessed during the follow-up period. The median duration of eltrombopag administration was 6 months (range 3-8 months). The initial response rate was 73.3%. Patients with megakaryocyte count ≥100/slide or Treg