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Received:April 14, 2021 Published Online:May 20, 2021
Received:April 14, 2021 Published Online:May 20, 2021
中文摘要: 血友病A是一种以凝血因子Ⅷ(Factor Ⅷ,FⅧ)缺乏,临床表现为自发性或者损伤后过度出血的X染色体连锁隐性遗传病。血友病A目前主要治疗方式是替代性输注FⅧ,但对于输注的剂量、频次大多仍采用固定输注模式,并不能达到个体化治疗原则下的最优疗效。影响FⅧ替代治疗疗效的因素中,个体对FⅧ产品的药物代谢动力学(Pharmacokinetics,PK)差异是一个重要因素。临床对于PK指导下的个体化FⅧ替代治疗认知尚不充分,因此本文就FⅧ的PK特征、分析模型、临床应用场景与具体治疗方案制定进行分析,并为临床实践提供指导意见。
Abstract:Hemophilia A is an X- chromosome- linked recessive genetic disease that lacks coagulation factor Ⅷ (Factor Ⅷ,FⅧ) and is clinically manifested as spontaneous or excessive bleeding after injury.The current main treatment for hemophilia A is alternative infusion of FⅧ,but the fixed infusion mode is still used for the dosage and frequency of infusion,which cannot achieve the optimal curative effect under the principle of individualized treatment.Among the factors that affect the efficacy of FⅧ replacement therapy,the difference in the pharmacokinetics (PK) of FⅧ products by individuals is an important factor.The clinical understanding of individualized FⅧ replacement therapy under the guidance of PK is not sufficient.Therefore,this article reviews the PK characteristics,analysis models,clinical application scenarios and specific treatment plan formulation of FⅧ.
文章编号: 中图分类号:R554 文献标志码:A
基金项目:十三五”国家重点研发计划精准医学研究重点专项(2016YFC0901503);中国医学科学院医学与健康科技创新工程重大协同创新项目(2017-I2M-3-018,2016-I2M-1-002)
Author Name | Affiliation |
Hemophilia Treatment Center Collaborative Network of China(Writer:CHEN Zhen- ping,LIU Wei,CAI Li- sheng) |
Author Name | Affiliation |
Hemophilia Treatment Center Collaborative Network of China(Writer:CHEN Zhen- ping,LIU Wei,CAI Li- sheng) |
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