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2024/5 | Authorea try { document.documentElement.classList.add('js'); } catch (e) { } var _gaq = _gaq || []; _gaq.push(['_setAccount', 'G-8VDV14Y67G']); _gaq.push(['_trackPageview']); (function() { var ga = document.createElement('script'); ga.type = 'text/javascript'; ga.async = true; ga.src = ('https:' == document.location.protocol ? 'https://ssl' : 'http://www') + '.google-analytics.com/ga.js'; var s = document.getElementsByTagName('script')[0]; s.parentNode.insertBefore(ga, s); })(); Skip to main content Preprints Collections Wiley Open Research IET Open Research Ecological Society of Japan All Collections About About Authorea FAQs Contact Us Quick Search anywhere Search for preprint articles, keywords, etc. Search Search ADVANCED SEARCH SCROLL This is a preprint and has not been peer reviewed. Data may be preliminary. 12 November 2025 V1 Latest version Share on 2024/5 Authors : Guichi Zhou 0009-0007-4854-1462 , Liya He , Chunyan Wang , Yashuang Deng , Dong Peng 0000-0001-5906-3303 , Zhaonan Liu , Sixi Liu , Xiaoying Fu , Ying Wang , and Xiaoqin Feng 0000-0003-1166-0138 [email protected] Authors Info & Affiliations https://doi.org/10.22541/au.176296690.08033734/v1 179 views 102 downloads Contents Abstract Supplementary Material Information & Authors Metrics & Citations View Options References Figures Tables Media Share Abstract Background: Emicizumab reduces bleeding episodes in children with hemophilia A. This study aimed to better elucidate the effectiveness and safety of emicizumab prophylaxis in younger children with hemophilia A in South China. Methods: This multicenter observational study retrospectively analyzed data from children with hemophilia A with and without inhibitors treated with prophylactic emicizumab (June 2019 to May 2025). Individualized dosing was used. Bleeding rates were calculated from 183 days before (or from birth) to at least 6 months after switching to emicizumab. In the maintenance period, factor VIII was determined using bovine factor IXa and factor X in the factor VIII chromogenic Bethesda assay. Canadian Haemophilia Outcomes-Kids’ Life Assessment Tool score was used to assess health-related quality of life. Results: Among 26 patients (mean age 3.3 years), bleeding events at emicizumab initiation were reported in 16 patients (61.5%) during emicizumab treatment; most were trauma related. Bleeding events were significantly reduced after initiating or switching to emicizumab. The median every-7-day equivalent emicizumab maintenance dose was 0.81 mg/kg. The mean equivalent factor VIII level was 8.21% ± 3.74%, which positively correlated with emicizumab, independent of inhibitors. Comparable equivalent factor VIII levels and weekly emicizumab consumption were observed among patients with and without inhibitors after switching to emicizumab. Health-related quality of life improved after emicizumab (scores increased from 57.23 ± 16.63 to 69.79 ± 13.32). Safety was favorable, with no adverse events. Conclusions: While exploratory, the findings suggest low-dose emicizumab prophylaxis may be beneficial in young Chinese children with hemophilia A. Supplementary Material File (manuscript.doc) Download 68.65 KB Information & Authors Information Version history V1 Version 1 12 November 2025 Copyright This work is licensed under a Non Exclusive No Reuse License. Keywords hematology hemophilia medical pediatric hematology/oncology Authors Affiliations Guichi Zhou 0009-0007-4854-1462 Shenzhen Children's Hospital View all articles by this author Liya He Guangzhou Women and Children's Medical Center View all articles by this author Chunyan Wang Shenzhen Children's Hospital View all articles by this author Yashuang Deng Shenzhen Children's Hospital View all articles by this author Dong Peng 0000-0001-5906-3303 Shenzhen Children's Hospital View all articles by this author Zhaonan Liu University of Toronto View all articles by this author Sixi Liu Shenzhen Children's Hospital View all articles by this author Xiaoying Fu Shenzhen Children's Hospital View all articles by this author Ying Wang Shenzhen Children's Hospital View all articles by this author Xiaoqin Feng 0000-0003-1166-0138 [email protected] Southern Medical University Nanfang Hospital View all articles by this author Metrics & Citations Metrics Article Usage 179 views 102 downloads .FvxKWukQNSOunydq8rnd { width: 100px; } Citations Download citation Guichi Zhou, Liya He, Chunyan Wang, et al. 2024/5. Authorea . 12 November 2025. DOI: https://doi.org/10.22541/au.176296690.08033734/v1 If you have the appropriate software installed, you can download article citation data to the citation manager of your choice. Simply select your manager software from the list below and click Download. For more information or tips please see 'Downloading to a citation manager' in the Help menu . Format Please select one from the list RIS (ProCite, Reference Manager) EndNote BibTex Medlars RefWorks Direct import Tips for downloading citations document.getElementById('citMgrHelpLink').addEventListener('click', function() { popupHelp(this.href); return false; }); $(".js__slcInclude").on("change", function(e){ if ($(this).val() == 'refworks') $('#direct').prop("checked", false); $('#direct').prop("disabled", ($(this).val() == 'refworks')); }); View Options View options PDF View PDF Figures Tables Media Share Share Share article link Copy Link Copied! Copying failed. 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