HETROMBOPAG demonstrates high response rates and a favourable safety profile in paediatric immune thrombocytopenia, supporting its role as an effective second line treatment option when first line therapy fails. Findings from a cohort of children treated with this thrombopoietin receptor agonist provide important clinical insight into outcomes that have previously been under explored in paediatric populations.
Efficacy Outcomes in Paediatric Immune Thrombocytopenia
Immune thrombocytopenia is an autoimmune disorder characterised by reduced platelet counts in both children and adults. This study evaluated 93 paediatric patients with immune thrombocytopenia who received hetrombopag as second line therapy. Multiple clinical outcomes were assessed, including complete response, response, overall response, durable response, relapse, and treatment free response. The complete response rate was 61.3%, while a further 15.1% achieved a response, resulting in an overall response rate of 76.3%. No response was observed in 23.7% of patients. Durable responses were achieved in 76.1% of responders, indicating sustained platelet recovery during follow up.
Treatment Free Response and Disease Duration
Treatment free response was achieved in 52.1% of patients, highlighting the potential for prolonged remission after discontinuation of therapy. Outcomes differed according to disease duration. Patients with newly diagnosed immune thrombocytopenia experienced higher treatment free response rates compared with those with persistent or chronic disease. These findings suggest that earlier use of hetrombopag may offer greater long term benefit in selected paediatric patients.
Switching from Other Thrombopoietin Receptor Agonists
The study also examined outcomes in a small subgroup of patients who had previously received other thrombopoietin receptor agonists. Among nine patients who switched to hetrombopag, seven achieved an overall response during initial treatment. Of these, three achieved a complete response and four achieved a response. This observation indicates that hetrombopag may retain efficacy even after prior exposure to alternative agents within the same therapeutic class.
Safety Profile of Hetrombopag
Hetrombopag was generally well tolerated. Adverse events were reported in 37.6% of patients, and no serious adverse events were observed. These safety findings are particularly relevant in paediatric care, where long term tolerability is a key consideration.
Overall, the data support hetrombopag as a safe and effective treatment option for paediatric immune thrombocytopenia, offering meaningful response rates and the potential for treatment free remission.
Reference
Wang J-H et al. Long-term efficacy and safety of hetrombopag in paediatric patients with immune thrombocytopenia. BJ Haem. 2026; https://doi.org/10.1111/bjh.70309.
