{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,26]],"date-time":"2026-10-26T04:16:34Z","timestamp":1792988194570,"version":"4.5.0"},"reference-count":21,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2024,2,16]],"date-time":"2024-02-16T00:00:00Z","timestamp":1708041600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["81970111"],"award-info":[{"award-number":["81970111"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Br J Haematol"],"published-print":{"date-parts":[[2024,5]]},"abstract":"<jats:title>Summary<\/jats:title><jats:p>Avatrombopag (AVA) is a novel thrombopoietin receptor agonist (TPO\u2010RA) that has been recently approved as a second\u2010line therapy for immune thrombocytopenia (ITP) in adults; however, its safety and efficacy data in children are lacking. Here, we demonstrated the efficacy and safety of AVA as second\u2010line therapy in children with ITP. A multicentre, retrospective, observational study was conducted in children with persistent or chronic ITP who did not respond to or relapsed from previous treatment and were treated with AVA for at least 12\u2009weeks between August 2020 and December 2022. The outcomes were the responses (defined as achieving a platelet count \u226530\u2009\u00d7\u200910<jats:sup>9<\/jats:sup>\/L, twofold increase in platelet count from baseline and absence of bleeding), including rapid response within 4\u2009weeks, sustained response at weeks 12 and 24, bleeding control and adverse events (AEs). Thirty\u2010four (18 males) patients with a mean age of 6.3 (range: 1.9\u201315.3) years were enrolled. The median number of previous treatment types was four (range: 1\u20136), and 41.2% patients switched from other TPO\u2010RAs. Within 4\u2009weeks, overall response (OR) was achieved in 79.4% patients and complete response (CR, defined as a platelet count \u2265100\u2009\u00d7\u200910<jats:sup>9<\/jats:sup>\/L and the absence of bleeding) in 67.7% patients with a median response time of 7 (range: 1\u201327) days. At 12\u2009weeks, OR was achieved in 88.2%, CR in 76.5% and sustained response in 44% of patients. At 24\u2009weeks, 22\/34 (64.7%) patients who achieved a response and were followed up for 24\u2009weeks were evaluated; 12\/22 (54.55%) achieved a sustained response. During AVA therapy, median platelet counts increased by week 1 and were maintained throughout the treatment period. The proportion of patients with grade 1\u20133 bleeding decreased from 52.95% at baseline to 2.94% at 12\u2009weeks, while concomitant ITP medications decreased from 36.47% at baseline to 8.82% at 12\u2009weeks, with only 9 (26.47%) patients receiving rescue therapy 23 times within 12\u2009weeks. There were 61.8% patients with 59 AEs: 29.8% with Common Terminology Criteria for Adverse Events grade 1 and the rest with grade 2. These findings show that AVA could achieve a rapid and sustained response in children with persistent or chronic ITP as a second\u2010line treatment, with good clinical bleeding control and reduction of concomitant ITP therapy, without significant AEs.<\/jats:p>","DOI":"10.1111\/bjh.19342","type":"journal-article","created":{"date-parts":[[2024,2,16]],"date-time":"2024-02-16T09:48:26Z","timestamp":1708076906000},"page":"1958-1965","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":16,"title":["Efficacy and safety of avatrombopag in Chinese children with persistent and chronic primary immune thrombocytopenia: A multicentre observational retrospective study in China"],"prefix":"10.1111","volume":"204","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7363-5101","authenticated-orcid":false,"given":"Zhifa","family":"Wang","sequence":"first","affiliation":[{"name":"Beijing Key Laboratory of Pediatric Hematology\u2010Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Hemophilia Comprehensive Care Centre, Hematology Centre, National Centre for Children's Health, Ministry of Education, Beijing Children's Hospital Capital Medical University  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6117-4560","authenticated-orcid":false,"given":"Aijun","family":"Zhang","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Qilu Hospital of Shandong University  Jinan China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhongjin","family":"Xu","sequence":"additional","affiliation":[{"name":"Department of Hematology Jiangxi Provincial Children's Hospital  Nanchang China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nan","family":"Wang","sequence":"additional","affiliation":[{"name":"Department of Pharmacy, Beijing Children's Hospital Capital Medical University  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7624-9430","authenticated-orcid":false,"given":"Jialu","family":"Zhang","sequence":"additional","affiliation":[{"name":"Beijing Key Laboratory of Pediatric Hematology\u2010Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Hemophilia Comprehensive Care Centre, Hematology Centre, National Centre for Children's Health, Ministry of Education, Beijing Children's Hospital Capital Medical University  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jinxi","family":"Meng","sequence":"additional","affiliation":[{"name":"Beijing Key Laboratory of Pediatric Hematology\u2010Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Hemophilia Comprehensive Care Centre, Hematology Centre, National Centre for Children's Health, Ministry of Education, Beijing Children's Hospital Capital Medical University  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shuyue","family":"Dong","sequence":"additional","affiliation":[{"name":"Beijing Key Laboratory of Pediatric Hematology\u2010Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Hemophilia Comprehensive Care Centre, Hematology Centre, National Centre for Children's Health, Ministry of Education, Beijing Children's Hospital Capital Medical University  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jingyao","family":"Ma","sequence":"additional","affiliation":[{"name":"Beijing Key Laboratory of Pediatric Hematology\u2010Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Hemophilia Comprehensive Care Centre, Hematology Centre, National Centre for Children's Health, Ministry of Education, Beijing Children's Hospital Capital Medical University  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yu","family":"Hu","sequence":"additional","affiliation":[{"name":"Beijing Key Laboratory of Pediatric Hematology\u2010Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Hemophilia Comprehensive Care Centre, Hematology Centre, National Centre for Children's Health, Ministry of Education, Beijing Children's Hospital Capital Medical University  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Juntao","family":"Ouyang","sequence":"additional","affiliation":[{"name":"Hematologic Disease Laboratory, Hematology Centre, Beijing Key Laboratory of Pediatric Hematology\u2010Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, National Centre for Children's Health, Ministry of Education, Beijing Pediatric Research Institute, Beijing Children's Hospital Capital Medical University  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhenping","family":"Chen","sequence":"additional","affiliation":[{"name":"Hematologic Disease Laboratory, Hematology Centre, Beijing Key Laboratory of Pediatric Hematology\u2010Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, National Centre for Children's Health, Ministry of Education, Beijing Pediatric Research Institute, Beijing Children's Hospital Capital Medical University  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Qi","family":"An","sequence":"additional","affiliation":[{"name":"Department of Hematology Xuzhou Children's Hospital, Xuzhou Medical University  Xuzhou China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0048-2658","authenticated-orcid":false,"given":"Xiaoling","family":"Cheng","sequence":"additional","affiliation":[{"name":"Department of Pharmacy, Beijing Children's Hospital Capital Medical University  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4030-209X","authenticated-orcid":false,"given":"Runhui","family":"Wu","sequence":"additional","affiliation":[{"name":"Beijing Key Laboratory of Pediatric Hematology\u2010Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Hemophilia Comprehensive Care Centre, Hematology Centre, National Centre for Children's Health, Ministry of Education, Beijing Children's Hospital Capital Medical University  Beijing 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