Treatment with eltrompobag appears to lead to a sustained response after removing treatment in children with immune thrombocytopenic purpura (ITP), according to a recently published study in the British Journal of Hematology.

Although ITP has been associated with a high rate of spontaneous remission in pediatric patients, approximately 35% of children with the condition develop persistent chronic ITP with severe complications, the researchers noted.

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Eltrompobag, a thrombopoietin receptor agonist, has been shown to significantly increase platelet production leading to a sustained response to treatment in a significant percentage of adult patients. However, there is scarce literature regarding its efficacy in the pediatric population, they added.

Therefore, the authors aimed to analyze the efficacy of eltrompobag as well as the percentage of patients achieving sustained response to treatment through a multicenter, retrospective study. The study included 143 patients.

All of the included patients were 18 years of age or under and had a history of not achieving a sustained response after first-line therapy with glucocorticosteroids or intravenous immunoglobulins and had received treatment exclusively with eltrompobag for at least 2 months. The sustained response off treatment was defined as platelet levels over 30.000/ml after 6 months off treatment.

Results revealed that 43% of patients achieved a complete response, defined a platelet level over 100.000/ ml, and over 20 percent had a response with platelet levels between 30.000/ml and 100.000/ml. Over 70% of patients achieved sustained response off treatment. Approximately 40 patients reported mild adverse effects to treatment; no severe adverse effects were reported.

The authors noted that patients that achieved sustained response to treatment tended to have an earlier beginning of treatment as well as a longer duration and slower reduction.

“Further studies of good design are needed to evaluate which patients are likely to achieve sustained response off treatment after tapering and withdrawal of eltrompobag,” the authors wrote.


Wang Z, Wang L, Liu Y, et al. Sustained response off treatment in ELTROMBOPAG for children with persistent/chronic primary immune thrombocytopenia: a multicentre observational retrospective study in China. Br J Haematol. Published online April, 20 2023. doi:10.1111/bjh.18842