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BOSTON,January 25,2026 – A new treatment is offering hope to children battling a rare bleeding disorder. Hetrombopag, a medication designed to boost platelet production, demonstrated a remarkable 76.3% overall response rate in pediatric patients with immune thrombocytopenia, a condition where the body mistakenly attacks its own platelets.
A Second Chance for Young Patients
Hetrombopag shows promise as a safe and effective option when initial treatments fail, potentially reducing the need for long-term steroid use.
- hetrombopag achieved a 61.3% complete response rate in children with immune thrombocytopenia.
- Over half (52.1%) of patients experienced treatment-free remission after stopping the medication.
- The drug was generally well-tolerated, with no serious adverse events reported.
- Patients newly diagnosed with the condition saw the highest rates of lasting remission.
Immune thrombocytopenia (ITP) affects both children and adults, causing dangerously low platelet counts and increasing the risk of bleeding. Current treatments often rely on steroids, which can have significant side effects, especially with prolonged use. This new research, published in the BJ Haem journal, focuses on hetrombopag as a second-line therapy for children whose ITP doesn’t respond to initial treatments.
How Effective is Hetrombopag?
Researchers evaluated 93 pediatric patients with ITP who received hetrombopag after other treatments had failed. The results were
