{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,7]],"date-time":"2026-07-07T00:29:15Z","timestamp":1783384155146,"version":"3.54.6"},"reference-count":26,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2024,9,12]],"date-time":"2024-09-12T00:00:00Z","timestamp":1726099200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2024,9,12]],"date-time":"2024-09-12T00:00:00Z","timestamp":1726099200000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"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"}]},{"DOI":"10.13039\/501100012166","name":"National Key Research and Development Program of China","doi-asserted-by":"publisher","award":["2023YFC2706100"],"award-info":[{"award-number":["2023YFC2706100"]}],"id":[{"id":"10.13039\/501100012166","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":[[2024,12]]},"abstract":"<jats:title>Summary<\/jats:title>\n                  <jats:p>The efficacy and safety of recombinant human thrombopoietin (rhTPO) in children and adolescent patients with chronic primary immune thrombocytopenia (ITP) remains unclear. A multicentre, randomized, double\u2010blind, placebo\u2010controlled phase III trial was performed. Patients aged 6\u201317\u2009years, diagnosed with ITP and resistant or relapsed to corticosteroid treatment were included. For the trial, part 1 was exploratory and part 2 was the main analysis, with part 1 determining whether part 2 was stratified by age. Patients in part 1 were treated with rhTPO (the 6\u2010 to 11\u2010\/12\u2010 to 17\u2010year\u2010old groups; 1:1). Patients in part 2 were randomized (3:1) to receive either rhTPO treatment or placebo. Patients received rhTPO or placebo at a dose of 300\u2009U\/kg once daily for up to 14\u2009days. A total of 68 patients were included [part 1 (12 patients), part 2 (56 patients)]. The total response rate (TRR) in part 1 was 50.0% (95% CI: 21.09%\u201378.91%). For part 2, the TRR was 58.5% (95% CI: 42.11%\u201373.68%) and 13.3% (95% CI: 1.66%\u201340.46%) in the rhTPO and placebo groups (FAS) respectively. The difference in TRR between the rhTPO group and placebo group was 45.2% (95% CI: 22.33%\u201368.08%) and 44.6% (95% CI: 21.27%\u201367.85%) on the FAS and per\u2010protocol set (PPS), respectively, which indicates the superiority of rhTPO treatment.<\/jats:p>","DOI":"10.1111\/bjh.19761","type":"journal-article","created":{"date-parts":[[2024,9,13]],"date-time":"2024-09-13T00:54:29Z","timestamp":1726188869000},"page":"2403-2413","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["Efficacy and safety of recombinant human thrombopoietin for the treatment of chronic primary immune thrombocytopenia in children and adolescents: A multicentre, randomized, double\u2010blind, placebo\u2010controlled phase\n                    <scp>III<\/scp>\n                    trial"],"prefix":"10.1111","volume":"205","author":[{"given":"Jingyao","family":"Ma","sequence":"first","affiliation":[{"name":"Department of Hematology, National Center for Children's Health Beijing Children's Hospital, Capital Medical University  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaoli","family":"Zhang","sequence":"additional","affiliation":[{"name":"Department of Pediatrics The First Affiliated Hospital, Sun Yat\u2010Sen University  Guangzhou China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Libo","family":"Zhao","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\/0000-0002-1644-5325","authenticated-orcid":false,"given":"Xiaoyan","family":"Wu","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Union Hospital, Tongji Medical College, Huazhong University of Science and Technology  Wuhan China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yanhua","family":"Yao","sequence":"additional","affiliation":[{"name":"Department of Hematology Children's Hospital of Soochow University  Suzhou China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wei","family":"Liu","sequence":"additional","affiliation":[{"name":"Department of Hematology Oncology Children's Hospital Affiliated of Zhengzhou University  Zhengzhou China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaohuan","family":"Wang","sequence":"additional","affiliation":[{"name":"Department of Hematology Children's Hospital of Shanxi  Taiyuan China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiuli","family":"Ju","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Qilu Hospital of Shandong University  Jinan China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaodong","family":"Shi","sequence":"additional","affiliation":[{"name":"Department of Hematology Capital Institute of Pediatrics  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lirong","family":"Sun","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Affiliated Hospital of Qingdao University  Qingdao China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lili","family":"Zheng","sequence":"additional","affiliation":[{"name":"Shenyang Sunshine Pharmaceuticals CO. Ltd.  Shenyang China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shu","family":"Liu","sequence":"additional","affiliation":[{"name":"Shenyang Sunshine Pharmaceuticals CO. Ltd.  Shenyang China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jun","family":"Qian","sequence":"additional","affiliation":[{"name":"Shenyang Sunshine Pharmaceuticals CO. Ltd.  Shenyang China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0007-4979-6942","authenticated-orcid":false,"given":"Runhui","family":"Wu","sequence":"additional","affiliation":[{"name":"Department of Hematology, National Center for Children's Health Beijing Children's Hospital, Capital Medical University  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,9,12]]},"reference":[{"key":"e_1_2_13_2_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMcp1810479"},{"key":"e_1_2_13_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.autrev.2017.04.012"},{"key":"e_1_2_13_4_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2003-08-2672"},{"key":"e_1_2_13_5_1","doi-asserted-by":"publisher","DOI":"10.3390\/jcm6020016"},{"key":"e_1_2_13_6_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13045-023-01401-z"},{"key":"e_1_2_13_7_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2019000966"},{"key":"e_1_2_13_8_1","doi-asserted-by":"publisher","DOI":"10.1080\/09537104.2020.1745168"},{"key":"e_1_2_13_9_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2017-09-742353"},{"key":"e_1_2_13_10_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2019.218883"},{"key":"e_1_2_13_11_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2141.2006.06215.x"},{"key":"e_1_2_13_12_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2007-05-088500"},{"key":"e_1_2_13_13_1","doi-asserted-by":"publisher","DOI":"10.1054\/blre.2001.0184"},{"key":"e_1_2_13_14_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40265-019-01159-0"},{"key":"e_1_2_13_15_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2009-06-225565"},{"key":"e_1_2_13_16_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2017-01-761262"},{"key":"e_1_2_13_17_1","doi-asserted-by":"publisher","DOI":"10.1007\/s12185-012-1124-8"},{"key":"e_1_2_13_18_1","doi-asserted-by":"publisher","DOI":"10.1080\/09537104.2023.2271568"},{"key":"e_1_2_13_19_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.25989"},{"key":"e_1_2_13_20_1","first-page":"1059","article-title":"Diagnosis and treatment guideline of primary immune thrombocytopenia in children (2019) [in Chinese]","volume":"17","author":"PRC, N.H.C.o.t","year":"2019","journal-title":"Clin Educ Gen Pract"},{"key":"e_1_2_13_21_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.iref.2020.12.026"},{"key":"e_1_2_13_22_1","first-page":"511","article-title":"Efficacy and safety of rhTPO in the treatment of pediatric primary immune thrombocytopenia","volume":"36","author":"Huang Y","year":"2015","journal-title":"Zhonghua Xue Ye Xue Za Zhi"},{"key":"e_1_2_13_23_1","doi-asserted-by":"publisher","DOI":"10.1186\/2008-2231-20-7"},{"key":"e_1_2_13_24_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2015-07-659656"},{"key":"e_1_2_13_25_1","doi-asserted-by":"publisher","DOI":"10.1080\/09537104.2022.2157806"},{"key":"e_1_2_13_26_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1387-2656(06)12008-6"},{"key":"e_1_2_13_27_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2018.212845"}],"container-title":["British Journal of Haematology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/bjh.19761","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1111\/bjh.19761","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/bjh.19761","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,2,14]],"date-time":"2026-02-14T22:10:40Z","timestamp":1771107040000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/bjh.19761"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,9,12]]},"references-count":26,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2024,12]]}},"alternative-id":["10.1111\/bjh.19761"],"URL":"https:\/\/doi.org\/10.1111\/bjh.19761","archive":["Portico"],"relation":{},"ISSN":["0007-1048","1365-2141"],"issn-type":[{"value":"0007-1048","type":"print"},{"value":"1365-2141","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,9,12]]},"assertion":[{"value":"2024-07-08","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-08-28","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-09-12","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}