{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T14:02:51Z","timestamp":1790863371330,"version":"4.1.0"},"reference-count":18,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2024,6,3]],"date-time":"2024-06-03T00:00:00Z","timestamp":1717372800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"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"}]},{"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":[[2024,7]]},"abstract":"<jats:title>Summary<\/jats:title><jats:p>Chronic refractory primary immune thrombocytopenia (CRITP) is currently defined as refractory to multiple therapeutic of second\u2010line agents with or without splenectomy, faced with the threat of severe bleeding and challenging to obtain effective treatment. Although stable and effective drug therapy is needed, it is tough to find one. Daratumumab (Dara), an anti\u2010CD38 monoclonal antibody presented the target cloned plasma cells in multiple myeloma, has also been reported to be effective in refractory autoimmune cytopenia in some case or series reports and ongoing clinical trials for adult patients with CRITP. Here, we report the early and durable response of Dara combination with avatrombopag in three CRITP patients (2 male and 1 female aged 12, 5 and 7\u2009years, respectively) in our centre, with a follow\u2010up period of more than 25\u2009weeks. Before Dara, the duration of immune thrombocytopenia was 9, 1.4 and 4\u2009years, respectively, a baseline platelet count of 4, 6, 9\u2009\u00d7\u200910<jats:sup>9<\/jats:sup>\/L, the bleeding score was all above level 2 and the number of previous drugs was &gt;3. The time to response (R: Plt \u226530\u2009\u00d7\u200910<jats:sup>9<\/jats:sup>\/L with at least a twofold increase in the baseline count) of Dara was on Day 45, 6 and 4 and achieved complete response (CR: Plt \u2265100\u2009\u00d7\u200910<jats:sup>9<\/jats:sup>\/L) on Day 51, 6 and 8, the sustained response (SR: Plt &gt;30\u2009\u00d7\u200910<jats:sup>9<\/jats:sup>\/L following Dara at \u226575% of the platelet count assessment at follow\u2010up end\u2010point since the patient achieved response) was 48, 175 and 204\u2009days with the follow\u2010up time of 39.1, 25.9 and 29.7\u2009weeks. The bleeding score decreased from grade 3 to grade 0 during follow\u2010up. No significant treatment\u2010related adverse events were found during follow\u2010up. Dara combination with avatrombopag may be a safe and efficacious therapy for children with CRITP, but it needs to be further explored.<\/jats:p>","DOI":"10.1111\/bjh.19553","type":"journal-article","created":{"date-parts":[[2024,6,4]],"date-time":"2024-06-04T01:58:40Z","timestamp":1717466320000},"page":"300-305","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["The early and rapid response to daratumumab in children with chronic refractory immune thrombocytopenia from a referral single centre of China"],"prefix":"10.1111","volume":"205","author":[{"given":"Yu","family":"Hu","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 Center, Hematology Center, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7363-5101","authenticated-orcid":false,"given":"Zhifa","family":"Wang","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 Center, Hematology Center, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, 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 Center, Hematology Center, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University  Beijing 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"}]},{"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 Center, Hematology Center, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, 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 Center, Hematology Center, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhenping","family":"Chen","sequence":"additional","affiliation":[{"name":"Hematologic Disease Laboratory, Hematology Center, Beijing Key Laboratory of Pediatric Hematology Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Pediatric Research Institute, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University  Beijing 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 Center, Hematology Center, Ministry of Education, Beijing Children's Hospital, National Center for Children's Health, Capital Medical University  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,6,3]]},"reference":[{"key":"e_1_2_13_2_1","doi-asserted-by":"publisher","DOI":"10.1002\/ped4.12305"},{"key":"e_1_2_13_3_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2019003599"},{"key":"e_1_2_13_4_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2016-03-603365"},{"key":"e_1_2_13_5_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.17872"},{"key":"e_1_2_13_6_1","doi-asserted-by":"publisher","DOI":"10.1007\/s12185-022-03527-1"},{"key":"e_1_2_13_7_1","doi-asserted-by":"publisher","DOI":"10.7326\/0003-4819-146-1-200701020-00007"},{"key":"e_1_2_13_8_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2141.2004.04889.x"},{"key":"e_1_2_13_9_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2008-01-131029"},{"key":"e_1_2_13_10_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2011-11-393975"},{"key":"e_1_2_13_11_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2019.243543"},{"key":"e_1_2_13_12_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2021005124"},{"key":"e_1_2_13_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(81)90441-4"},{"key":"e_1_2_13_14_1","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2019.02458"},{"key":"e_1_2_13_15_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2021.279232"},{"key":"e_1_2_13_16_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2021-151410"},{"key":"e_1_2_13_17_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00277-022-05035-y"},{"key":"e_1_2_13_18_1","doi-asserted-by":"publisher","DOI":"10.1172\/JCI153580"},{"key":"e_1_2_13_19_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2023-188664"}],"container-title":["British Journal of Haematology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/bjh.19553","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,7,23]],"date-time":"2025-07-23T09:10:34Z","timestamp":1753261834000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/bjh.19553"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,6,3]]},"references-count":18,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2024,7]]}},"alternative-id":["10.1111\/bjh.19553"],"URL":"https:\/\/doi.org\/10.1111\/bjh.19553","archive":["Portico"],"relation":{},"ISSN":["0007-1048","1365-2141"],"issn-type":[{"value":"0007-1048","type":"print"},{"value":"1365-2141","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,6,3]]},"assertion":[{"value":"2024-02-12","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-05-13","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-06-03","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}