Objective To investigate the effect parameters on FⅧ pharmacokinetic parameters in pediatric patients with hemophilia A(HA),draw up individualized preventive treatment plans,and observe clinical efficacy.Methods Seventy-three HA patients treated in Cheng Du children Hemophilia Center from May 2019 to November 2023 were enrolled.Individualized preventive treatment was developed,and thirty-five patients who were regularly followed up 1 year,the annual joint bleeding rate(AJBR),joint ultrasound score and the trough FⅧ level,mean annual total factor consumption were observed and compared before and after treatment.Results The FⅧ half-life of patients with type O blood were significantly shorter than those with other blood types.vwf activity is positively correlated with FⅦ half-life.With age increased,FⅧ half-life were prolonged.A prophylaxis plan for thirty-five was formulated under the guidance of PK parameters.After 1 year of follow-up,the mean AJBR dropped from 1.0(0-7)to 0(0-5),and the average ultrasound score dropped from(5.3±2.2)to(3.7±1.6).Mean total factor consumption increased from(2417.9tal fa)IU/(kg.year)to(3150.6tal fa)IU/(kgg0.6t),After PK-tailored prophylaxis,14 patients adjusted the treatment plan,7 patients increased the dosage,7 patients increased infusion frequency,and the number of patients with zero AJBR increased from 17 to 25.Conclusions PK-tailored prophylaxis can optimize individualized therapy in patients with hemophilia A.
维普
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