Chinese Expert Consensus on Pharmacokinetics Guided Treatment for Hemophilia A / 罕见病研究

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.