{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,24]],"date-time":"2025-10-24T13:19:56Z","timestamp":1761311996330,"version":"3.40.5"},"reference-count":19,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2023,9,17]],"date-time":"2023-09-17T00:00:00Z","timestamp":1694908800000},"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":["82270133"],"award-info":[{"award-number":["82270133"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2023,11]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objectives<\/jats:title><jats:p>To assess current treatment\u2010related outcomes for children with severe and moderate haemophilia A (cHA) in China.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>This cross\u2010section Patient Report Outcome (PRO) report collected PRO data of severe and moderate cHAs registered in the 'Hemophilia Home Care Center' database (<jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"http:\/\/web.bjxueyou.cn\">http:\/\/web.bjxueyou.cn<\/jats:ext-link>) between January 2021 and November 2022. Data included records of bleeding, activities, and concentrates consumption. All patients had a confirmed diagnosis of moderate or severe haemophilia A (FVIII: C \u2264 5%) and were\u00a0&lt;\u00a018 years old.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Among 1038 analysable cases, 9.6% of children with inhibitors had a higher rate of intracranial haemorrhage, dropout school rate, and higher FVIII consumption than children without inhibitors. Among 100 children with inhibitors, 36 patients were treated without immune tolerance induction (ITI), 14 patients with irregular treatment and 50 patients received ITI. Children with ITI had a lower ABR (2.4 (0,6.6) vs. 13.4 (9.5, 26.6), <jats:italic>p<\/jats:italic>&lt;.001) and AJBR (0 (0, 3.1) vs. 8.9 (1.6, 19.3), <jats:italic>p<\/jats:italic>\u00a0&lt;\u00a0.001) compared to those without ITI. Among 938 children without inhibitors, 28.5% received on\u2010demand treatment and 71.5% received prophylaxis. Of 528 children with 1343.8 (1050.4, 2922.9)IU\/kg\/year median FVIII consumption, 43.0% received low\u2010dose, 43.2% received intermediate\u2010dose, and 13.8% received high\u2010dose regimen; these children with prophylaxis had a lower ABR (3.1 (0, 10.7) vs. 12.8 (2.4, 45.5), <jats:italic>p<\/jats:italic>\u00a0&lt;\u00a0.001), AJBR (0.5 (0, 3.9) vs. 3.0 (0, 12.0), <jats:italic>p<\/jats:italic>\u00a0&lt;\u00a0.001) and disability rate (9.0% vs.18.5%, <jats:italic>p<\/jats:italic>\u00a0=\u00a0.032) compared to children who received on\u2010demand treatment.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>The high rate of drop\u2010out of school and disability still present a huge gap to meet the needs in China. It is necessary to improve the level of medical accessibility and medicine affordability and strengthen the patient\/parent's education in China.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14863","type":"journal-article","created":{"date-parts":[[2023,9,18]],"date-time":"2023-09-18T06:49:47Z","timestamp":1695019787000},"page":"1467-1474","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Treatment\u2010related outcome from patient report outcome (PRO) of children with severe and moderate hemophilia A in China: An analysis report of registration data from patient organization \u201chemophilia home care center\u201d"],"prefix":"10.1111","volume":"29","author":[{"given":"Tao","family":"Guan","sequence":"first","affiliation":[{"name":"Beijing Hemophilia Home Care Centre  Beijing China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9987-9099","authenticated-orcid":false,"given":"Di","family":"Ai","sequence":"additional","affiliation":[{"name":"Hematology Centre Beijing Key Laboratory of Paediatric Haematology Oncology National Key Discipline of Paediatrics Capital Medical University; Key Laboratory of Major Diseases in Children Ministry of Education Beijing Childrenationrsity; Capital Medical University National Centre for Childrenal Univer  Beijing China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nan","family":"Zhao","sequence":"additional","affiliation":[{"name":"Beijing Hemophilia Home Care Centre  Beijing China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Guoqing","family":"Liu","sequence":"additional","affiliation":[{"name":"Hematology Centre Beijing Key Laboratory of Paediatric Haematology Oncology National Key Discipline of Paediatrics Capital Medical University; Key Laboratory of Major Diseases in Children Ministry of Education Beijing Childrenationrsity; Capital Medical University National Centre for Childrenal Univer  Beijing China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Wanru","family":"Yao","sequence":"additional","affiliation":[{"name":"Hematology Centre Beijing Key Laboratory of Paediatric Haematology Oncology National Key Discipline of Paediatrics Capital Medical University; Key Laboratory of Major Diseases in Children Ministry of Education Beijing Childrenationrsity; Capital Medical University National Centre for Childrenal Univer  Beijing China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5273-145X","authenticated-orcid":false,"given":"Zhenping","family":"Chen","sequence":"additional","affiliation":[{"name":"Hematologic Disease Laboratory Beijing Paediatric Research Institute Beijing Childrenute Laborat Capital Medical University National Centre for Childrenal Univer  Beijing China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6290-9074","authenticated-orcid":false,"given":"Zhenping","family":"Li","sequence":"additional","affiliation":[{"name":"Hematology Centre Beijing Key Laboratory of Paediatric Haematology Oncology National Key Discipline of Paediatrics Capital Medical University; Key Laboratory of Major Diseases in Children Ministry of Education Beijing Childrenationrsity; Capital Medical University National Centre for Childrenal Univer  Beijing China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yaohan","family":"Zhou","sequence":"additional","affiliation":[{"name":"Hematology Centre Beijing Key Laboratory of Paediatric Haematology Oncology National Key Discipline of Paediatrics Capital Medical University; Key Laboratory of Major Diseases in Children Ministry of Education Beijing Childrenationrsity; Capital Medical University National Centre for Childrenal Univer  Beijing China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4030-209X","authenticated-orcid":false,"given":"Runhui","family":"Wu","sequence":"additional","affiliation":[{"name":"Hematology Centre Beijing Key Laboratory of Paediatric Haematology Oncology National Key Discipline of Paediatrics Capital Medical University; Key Laboratory of Major Diseases in Children Ministry of Education Beijing Childrenationrsity; Capital Medical University National Centre for Childrenal Univer  Beijing China"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2023,9,17]]},"reference":[{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14046"},{"key":"e_1_2_11_3_1","doi-asserted-by":"publisher","DOI":"10.1002\/rth2.12248"},{"key":"e_1_2_11_4_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13447"},{"key":"e_1_2_11_5_1","unstructured":"Thrombosis and Hemostasis Group Hematology Society of Chinese Medical Association Hemophilia Treatment Center Collaborative Network of China Zhonghua xue ye xue za zhi.2017;37(5):364\u2010370."},{"key":"e_1_2_11_6_1","doi-asserted-by":"publisher","DOI":"10.1111\/jth.12672"},{"key":"e_1_2_11_7_1","doi-asserted-by":"publisher","DOI":"10.1055\/s-0040-1713097"},{"key":"e_1_2_11_8_1","doi-asserted-by":"publisher","DOI":"10.1542\/peds.108.6.e105"},{"key":"e_1_2_11_9_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2016-02-683169"},{"key":"e_1_2_11_10_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14217"},{"key":"e_1_2_11_11_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14603"},{"key":"e_1_2_11_12_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2021011849"},{"key":"e_1_2_11_13_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.23957"},{"key":"e_1_2_11_14_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2006.01296.x"},{"key":"e_1_2_11_15_1","doi-asserted-by":"publisher","DOI":"10.1186\/s12913-015-1134-3"},{"key":"e_1_2_11_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.thromres.2016.10.015"},{"key":"e_1_2_11_17_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.17322"},{"key":"e_1_2_11_18_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10198-021-01339-4"},{"key":"e_1_2_11_19_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14029"},{"key":"e_1_2_11_20_1","doi-asserted-by":"publisher","DOI":"10.1016\/S2352-3026(19)30054-7"}],"container-title":["Haemophilia"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/hae.14863","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,1,19]],"date-time":"2024-01-19T11:20:19Z","timestamp":1705663219000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/hae.14863"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,9,17]]},"references-count":19,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2023,11]]}},"alternative-id":["10.1111\/hae.14863"],"URL":"https:\/\/doi.org\/10.1111\/hae.14863","archive":["Portico"],"relation":{},"ISSN":["1351-8216","1365-2516"],"issn-type":[{"type":"print","value":"1351-8216"},{"type":"electronic","value":"1365-2516"}],"subject":[],"published":{"date-parts":[[2023,9,17]]},"assertion":[{"value":"2023-02-18","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-09-01","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-09-17","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}