Study Overview and Patient Population
The analysis evaluated outcomes in 93 paediatric patients diagnosed with immune thrombocytopenia who received hetrombopag as second-line treatment. The study assessed multiple clinical endpoints, including platelet response, durability of response, relapse rates and treatment-free remission.
Children included in the cohort represented varying stages of disease duration, ranging from newly diagnosed cases to those with persistent and chronic immune thrombocytopenia. This allowed investigators to assess how disease stage influenced treatment outcomes.
High Response Rates Observed
The results showed encouraging efficacy across the cohort. A complete response, defined as restoration of platelet counts to target levels, was achieved in 61.3% of patients. An additional 15.1% experienced a partial response, resulting in an overall response rate of 76.3%.
Approximately 23.7% of patients did not respond to therapy. However, among those who achieved a response, the majority maintained stable platelet levels during follow-up, indicating sustained benefit from treatment.
Durable and Treatment-Free Responses
Durable responses were reported in 76.1% of responders, highlighting the ability of hetrombopag to provide ongoing platelet recovery. Notably, treatment-free response was achieved in 52.1% of patients, suggesting that more than half of treated children were able to maintain remission after discontinuing therapy.
Outcomes varied based on disease duration. Children with newly diagnosed immune thrombocytopenia demonstrated higher treatment-free response rates compared with those with persistent or chronic disease, indicating that earlier use of hetrombopag may offer greater long-term benefit.
Efficacy After Prior Therapy Failure
The study also explored outcomes in a small subgroup of patients who had previously been treated with other thrombopoietin receptor agonists. Among nine children who switched to hetrombopag, seven achieved an overall response during initial treatment.
Within this group, three patients achieved complete response while four achieved partial response. These findings suggest that hetrombopag may remain effective even in patients who have failed or discontinued other agents in the same drug class.
Safety and Tolerability in Children
Safety outcomes were favourable, an important consideration in paediatric care where long-term treatment effects are closely scrutinised. Adverse events were reported in 37.6% of patients, most of which were mild and manageable.
No serious adverse events were observed during the study period. The absence of severe safety concerns supports the suitability of hetrombopag for extended use in children requiring ongoing platelet support.
Clinical Implications
The findings provide valuable insight into an area that has historically been underexplored in paediatric haematology. With limited second-line options available for children with immune thrombocytopenia, the results position hetrombopag as a promising therapeutic choice.
