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Long-term efficacy and safety of prophylaxis with recombinant factor VIII in Chinese pediatric patients with hemophilia A: a multi-center, retrospective, non-interventional, phase IV (ReCARE) study

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单位: [1]Shenzhen Childrens Hosp, Dept Hematol & Oncol, Shenzhen, Peoples R China [2]Shandong Blood Ctr, Hemophilia Treatment Ctr, Jinan, Peoples R China [3]Beijing Union Med Coll Hosp, Dept Hematol, 1 Shuaifuyuan, Beijing 100730, Peoples R China [4]Capital Med Univ, Beijing Childrens Hosp, Hematol Oncol Ctr, Beijing, Peoples R China [5]HUST, Dept Hematol, Tongji Hosp, Tongji Med Coll, Wuhan, Peoples R China [6]Zhengjiang Univ, Sch Med, Childrens Hosp, Dept Hematol, Hangzhou, Zhejiang, Peoples R China [7]Southern Med Univ, Nan Fang Hosp, Dept Hematol, Guangzhou, Guangdong, Peoples R China [8]Chinese Acad Med Sci, Thrombosis & Hemostasis Ctr, Inst Haematol, Tianjin, Peoples R China [9]Chinese Acad Med Sci, Blood Dis Hosp, Tianjin, Peoples R China [10]Peking Union Med Coll, Tianjin, Peoples R China [11]Chengdu Women & Childrens Cent Hosp, Dept Hematol, Chengdu, Peoples R China [12]Nanjing Univ, Med Sch, Nanjing Drum Tower Hosp, Dept Hematol, Nanjing, Jiangsu, Peoples R China [13]Dalian Med Univ, Dalian Municipal Cent Hosp, Dept Hematol, Dalian, Peoples R China [14]Yangzhou Univ, Clin Med Sch, Dept Hematol, Northern Jiangsu Peoples Hosp, Yangzhou, Jiangsu, Peoples R China [15]Bayer HealthCare Pharmaceut Inc, Bayer Ctr, Beijing, Peoples R China
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关键词: Hemophilia A annualized bleeding rate Kogenate Pediatric Prophylaxis ReCARE rFVIII

摘要:
Background: The first recombinant factor VIII (rFVIII) product was launched in China in 2007. However, until now, no study has been conducted to describe the efficacy and safety of prophylaxis with rFVIII in Chinese pediatric patients with hemophilia A (HA). Objective: To summarize the efficacy and safety data on prophylaxis with rFVIII in Chinese pediatric patients with HA. Methods: ReCARE (Retrospective study in Chinese pediatric hemophilia A patients with rFVIII contained regular prophylaxis) was a retrospective study conducted in 12 hemophilia treatment centers (HTCs) across China. The primary endpoints included reduction in annualized bleeding rate (ABR); the secondary endpoints included evaluation of joint function (number and sites of target joints) using Gilbert score and Hemophilia Joint Health Score (HJHS), quality of life (QoL) and factors affecting treatment choices. Safety assessment of rFVIII was also conducted. Results: We analyzed a total of 183 male pediatric patients (mean age, 7.14.23 years) who received prophylaxis between 1 November 2007 and 31 May 2013. Compared with baseline, prophylaxis with rFVIII significantly reduced overall annualized joint bleed rate (AJBR) (p<.001) and ABR (p<.001). Inhibitor formation was reported in 5 (2.7%) patients and hemarthrosis was reported in 1 patient. The mean number of target joints was positively related to age (p<.001) and weight (p=.003) at baseline. Responses from survey questionnaires reported that effective bleeding control, joint protection, improvement in quality of life, favorable medical insurance policies, and economic capability were reasons for choosing prophylaxis. Conclusion: Prophylaxis with rFVIII reduced bleeding and number of target joints, even with a low-dose regimen, in Chinese pediatric patients with HA. Other than the efficacy and safety, factors such as poor disease control, improved economic stability and stable financial support made prophylaxis as an attractive treatment option.

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出版当年[2016]版:
大类 | 3 区 医学
小类 | 3 区 医学:内科 3 区 医学:研究与实验
最新[2025]版:
大类 | 4 区 医学
小类 | 3 区 医学:内科 4 区 医学:研究与实验
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出版当年[2015]版:
Q1 MEDICINE, GENERAL & INTERNAL Q2 MEDICINE, RESEARCH & EXPERIMENTAL
最新[2023]版:
Q1 MEDICINE, GENERAL & INTERNAL Q3 MEDICINE, RESEARCH & EXPERIMENTAL

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第一作者单位: [1]Shenzhen Childrens Hosp, Dept Hematol & Oncol, Shenzhen, Peoples R China
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