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The benefit of low dose prophylaxis in the treatment of hemophilia: a focus on China

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机构: [1]Capital Med Univ, Hemophilia Work Grp, Hematol Oncol Ctr, Beijing Childrens Hosp, Beijing, Peoples R China; [2]Univ Ottawa, Childrens Hosp Eastern Ontario, Ottawa, ON, Canada
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关键词: China hemophilia children low dose prophylaxis treatment

摘要:
Introduction: Currently full dose prophylaxis is the standard of care in the treatment of hemophilia (World Federation of Hemophilia). However, the high costs prevent the use of standard or intermediate dose prophylaxis in China and other developing countries. Low dose prophylaxis would be a viable alternative treatment. At present global research data on the use of low dose prophylaxis is limited.Areas covered: Since 2007, China has been developing low dose prophylaxis as a high priority (90 % of moderate and severe hemophilia boys suffer joint disease by age 6 - 9). 11 studies were successfully conducted and published results showing evidence of the benefits of low dose prophylaxis to reduce joint bleeding. This new knowledge has been implemented into clinical practice in China. However the long-term outcome of arthropathy remains unclear and obstacles in execution exist.Expert commentary: In 2016, the first phenotype-based individualized prophylaxis study using four escalating low dose regimens on severe Chinese hemophilia A boys (China Individualized Prophylaxis Study (CHIP China)) launched. Using the previously published and imminent CHIP data, the goal for China is to establish an effective escalating low dose prophylaxis protocol for use in China as a standard of care.

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中科院(CAS)分区:
出版当年[2016]版
大类 | 3 区 医学
小类 | 4 区 血液学
最新[2023]版
大类 | 4 区 医学
小类 | 4 区 血液学
JCR分区:
出版当年[2015]版:
Q3 HEMATOLOGY
最新[2023]版:
Q2 HEMATOLOGY

影响因子: 最新[2023版] 最新五年平均 出版当年[2015版] 出版当年五年平均 出版前一年[2014版] 出版后一年[2016版]

第一作者:
第一作者机构: [1]Capital Med Univ, Hemophilia Work Grp, Hematol Oncol Ctr, Beijing Childrens Hosp, Beijing, Peoples R China;
通讯作者:
通讯机构: [1]Capital Med Univ, Hemophilia Work Grp, Hematol Oncol Ctr, Beijing Childrens Hosp, Beijing, Peoples R China;
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