Efficacy of Romiplostim in Children with Persistent or Chronic ITP
The effect of the thrombopoietin receptor agonist romiplostim in adult patients with immune thrombocytopenia (ITP) is well known. Equally important, however, are data on its treatment in children — presented by a study whose results were published in the journal Lancet.
Romiplostim in Children with ITP
The thrombopoietin receptor agonist romiplostim may be effective in the treatment of symptomatic children with persistent or chronic immune thrombocytopenia. Data published in Lancet evaluate its efficacy and safety in children with ITP lasting ≥ 6 months.
Study Methodology and Course
In the phase III double-blind study, children with ITP aged 1–17 years with an average platelet count ≤ 30 × 109/L (average of 2 measurements during the screening period) were enrolled. Patients were randomly assigned in a 2:1 ratio to treatment with romiplostim or placebo for 24 months. They were stratified by age (1–5 years, 6–11 years, 12–17 years) and by weekly dosage in the range of 1–10 μg/kg aiming to achieve a platelet count of 50–200 × 109/L.
The primary analysis included all randomized patients, while the safety analysis included patients who received at least one dose of the investigational product. The primary endpoint was a sustained platelet response, defined as ≥ 50 × 109/L platelets without receiving any rescue therapy in the previous 4 weeks for a duration of ≥ 6 out of the final 8 weeks (weeks 18–25).
Results
The study included 62 patients, of whom 42 received romiplostim and 20 placebo. A sustained platelet response was observed in 22 (52%) patients in the romiplostim group and in 2 (10%) patients receiving placebo (odds ratio [OR] 9.1; 95% confidence interval [CI] 1.9–43.2; p = 0.002). The rate of sustained platelet response was 38% in children aged up to 5 years, 56% in children aged 6–11 years, and 56% in children aged 12–17 years.
Adverse events were recorded in 1 (5%) patient in the placebo group and 10 (24%) children treated with romiplostim, with one case involving headaches and thrombocytosis, both associated with romiplostim administration. However, no patient discontinued treatment due to adverse events.
Conclusion
In children with chronic ITP, romiplostim treatment led to a high rate of platelet response, with no new safety risks identified. Long-term safety, efficacy, and remission duration studies in children with ITP are ongoing.
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Source: Trantino M. D., Bussel J. B., Blanchette V. S. et al. Romiplostim in children with immune thrombocytopenia: a phase 3, randomised, double-blind, placebo-controlled study. Lancet 2016; 388 (10039): 45–54, doi: 10.1016/S0140-6736(16)00279-8.
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