Blood Beijing Children's Hospital BeiJing, Beijing, China (People's Republic)
Background: Immune thrombocytopenia(ITP) is the most common hemorrhagic disease in children, which is mainly caused by increased destruction and decreased production of autologous platelets due to abnormal autoimmune tolerance. The current international guidelines recommend that the second-line therapy drugs for children with chronic ITP are thrombopoietic agents (TRAs) and rituximab. Efficacy of combination therapy has been reported. Our center used the escalating treatment strategy, including three steps: Step I (high-dose dexamethasone), Step II (rituximab), and Step III (eltrombopag).
Aims: The purpose of this study was to confirm the efficacy of escalating treatment strategy in the treatment of childhood CITP.
Methods: This was a single-center, retrospective cohort study. Chidren were divided into 2 groups according to the treatment regimen: the group A (escalating treatment strategy) and the group B (eltrombopag). We evaluated the therapeutic effect after 12 months of follow-up.
Results: A total of 58 cases (30 males and 28 females) were included. Overall response was achieved in 72.41% of patients in the group A vs 68.97% in the group B, and the complete response (CR) rate was 34.48% in the group A compared with 20.69% in the group B. There was no difference between two groups (p＞0.05).
Conclusion(s): This study suggests that escalating treatment strategy can achieve the similar efficacy as TRAs. It is an effective, safe and second-line treatment scheme suitable for Chinese patients. It is still necessary to further evaluate the feasibility of shortening the course of treatment and reducing the burden of treatment.
