Daijiworld Media Network – New Delhi
New Delhi, Jan 25: Hetrombopag has demonstrated high response rates along with a favourable safety profile in children suffering from immune thrombocytopenia (ITP), reinforcing its role as an effective second-line treatment option when first-line therapy fails, according to recent clinical findings.
The study evaluated 93 paediatric patients diagnosed with immune thrombocytopenia who were treated with hetrombopag after inadequate response to initial therapy. Immune thrombocytopenia is an autoimmune disorder marked by low platelet counts and an increased risk of bleeding.
Data revealed a complete response in 61.3% of patients, while an additional 15.1% showed a partial response, resulting in an overall response rate of 76.3%. However, 23.7% of the patients did not respond to the treatment. Notably, 76.1% of responders maintained durable platelet recovery during follow-up, highlighting the sustained efficacy of the drug.
The study also reported that more than half of the patients (52.1%) achieved treatment-free response, indicating prolonged remission even after discontinuation of therapy. Children with newly diagnosed ITP showed better outcomes compared to those with persistent or chronic disease, suggesting that early introduction of hetrombopag may offer improved long-term benefits.
In a subgroup analysis, nine patients who had previously received other thrombopoietin receptor agonists were switched to hetrombopag. Seven of them achieved an overall response, including three complete responses, indicating that hetrombopag may remain effective even after prior exposure to similar treatments.
On the safety front, hetrombopag was generally well tolerated. Adverse events were reported in 37.6% of patients, but no serious adverse events were observed, making it a promising option in paediatric care where long-term safety is a critical consideration.
The findings underscore hetrombopag’s potential as a reliable and safe therapeutic option for children with immune thrombocytopenia, offering meaningful platelet recovery and the possibility of sustained remission without continuous treatment.