{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,28]],"date-time":"2026-08-28T08:20:19Z","timestamp":1787905219298,"version":"build-2784847793"},"reference-count":20,"publisher":"Wiley","license":[{"start":{"date-parts":[[2026,8,28]],"date-time":"2026-08-28T00:00:00Z","timestamp":1787875200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2026,8,28]],"date-time":"2026-08-28T00:00:00Z","timestamp":1787875200000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Br J Haematol"],"abstract":"<jats:title>Summary<\/jats:title>\n                  <jats:p>\n                    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\u2010centre, prospective trial, 54 children with newly diagnosed SAA were randomized to receive standard immunosuppressive therapy (IST) alone (\n                    <jats:italic>n<\/jats:italic>\n                    \u2009=\u200927) or combined with HPAG (IST+HPAG,\n                    <jats:italic>n<\/jats:italic>\n                    \u2009=\u200927). The primary end\u2010point was complete response (CR) rate (CRR) at 6\u2009months. Baseline characteristics were balanced. At 6\u2009months, the CRRs were comparable (59.3% vs. 62.9%,\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.948) and overall response (OR) rates (ORRs) identical (85.2%) between HPAG+IST and IST groups. At 12\u2009months, ORRs remained identical (77.8%) and CRRs were 62.9% vs. 74.1% (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.875). Time to response showed no significant differences. In children older than 9\u2009years, the 3\u2010month CRR was significantly higher with IST+HPAG (44.4% vs. 27.3%,\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.049). Eight grade \u22653 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\u2010line IST did not significantly improve early or sustained haematological response rates in paediatric SAA. Routine upfront combination therapy is not warranted for most patients.\n                  <\/jats:p>","DOI":"10.1111\/bjh.70636","type":"journal-article","created":{"date-parts":[[2026,8,28]],"date-time":"2026-08-28T07:58:40Z","timestamp":1787903920000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["First\u2010line hetrombopag combined with immunosuppressive therapy for paediatric severe aplastic anaemia: A prospective randomized controlled trial"],"prefix":"10.1111","author":[{"given":"Lingling","family":"Fu","sequence":"first","affiliation":[{"name":"Department of Hematology Center National Center for Children's Health, Beijing Children's Hospital, Capital Medical University  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bixi","family":"Yang","sequence":"additional","affiliation":[{"name":"Department of Hematology Center National Center for Children's Health, Beijing Children's Hospital, Capital Medical University  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0009-0683-8393","authenticated-orcid":false,"given":"Ruixin","family":"Wang","sequence":"additional","affiliation":[{"name":"Department of Hematology Center National Center for Children's Health, Beijing Children's Hospital, Capital Medical University  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hongmin","family":"Li","sequence":"additional","affiliation":[{"name":"Department of Hematology Center National Center for Children's Health, Beijing Children's Hospital, Capital Medical University  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hui","family":"Chen","sequence":"additional","affiliation":[{"name":"Department of Clinical Laboratory Center National Center for Children's Health, Beijing Children's Hospital, Capital Medical University  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6759-075X","authenticated-orcid":false,"given":"Jie","family":"Ma","sequence":"additional","affiliation":[{"name":"Department of Hematology Center National Center for Children's Health, Beijing Children's Hospital, Capital Medical University  Beijing 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