First-line hetrombopag combined with immunosuppressive therapy for paediatric severe aplastic anaemia: A prospective randomized controlled trial
In brief
Hetrombopag adds no benefit to first-line immunosuppression in pediatric severe aplastic anemia
In a randomized trial of 54 children, the complete response rate at six months was essentially the same with or without hetrombopag (59% vs 63%), and overall response remained identical at 12 months. Only a modest early advantage appeared in kids older than nine, and serious side effects were manageable, suggesting routine upfront combination therapy is not justified.
- Journal
- British journal of haematology (Q1)
- Published
- 28 August 2026
- Study design
- Randomized controlled trial
- Evidence level
- Level 1, High (CEBM 1b)
- Authors
- Lingling Fu, Bixi Yang, Ruixin Wang, Hongmin Li, Hui Chen, Jie Ma
- PMID
- 42661530
- DOI
- 10.1111/bjh.70636
Why clinicians should know about it
- Picked for Hematology (top studies of the week, 11 October 2026): Hetrombopag + IST in pediatric severe aplastic anaemia
Abstract
Although the thrombopoietin receptor agonist hetrombopag (HPAG) improves response in adults with severe aplastic anaemia (SAA), its efficacy in paediatric patients remains unclear. In this single-centre, prospective trial, 54 children with newly diagnosed SAA were randomized to receive standard immunosuppressive therapy (IST) alone (n = 27) or combined with HPAG (IST+HPAG, n = 27). The primary end-point was complete response (CR) rate (CRR) at 6 months. Baseline characteristics were balanced. At 6 months, the CRRs were comparable (59.3% vs. 62.9%, p = 0.948) and overall response (OR) rates (ORRs) identical (85.2%) between HPAG+IST and IST groups. At 12 months, ORRs remained identical (77.8%) and CRRs were 62.9% vs. 74.1% (p = 0.875). Time to response showed no significant differences. In children older than 9 years, the 3-month CRR was significantly higher with IST+HPAG (44.4% vs. 27.3%, p = 0.049). Eight grade ≥3 adverse events (primarily elevated liver enzymes) occurred in the IST+HPAG group, all manageable without treatment discontinuation. Rates of clonal events were comparable (23.8% vs. 26.3%). Adding HPAG to first-line IST did not significantly improve early or sustained haematological response rates in paediatric SAA. Routine upfront combination therapy is not warranted for most patients.
Abstract as published, via PubMed.
For healthcare professionals. The summary is generated by AI from the published abstract, and the evidence level is assigned automatically from the study design on the Oxford CEBM hierarchy. Neither is medical advice. Read the full paper before changing practice.