Efficacy Of Prophylaxis With Different Doses Factor ? In Children With Severe Hemophilia A

ObjectiveTo study the efficacy of prophylaxis with different doses factor ? in children with severe hemophilia A by joint ultrasound and other evaluation methods.Methods38 children with severe hemophilia A were included in this study.Among them,13 cases received prophylaxis with low-dose factor ?(10U/kg,three times a week),8 cases received prophylaxis with intermediate-dose factor ?(15-30 U /kg,3 times a week),7 cases received prophylaxis with standard-dose factor ?(25-40U/kg,3 times a week),and 10 cases received on-demand treatment with factor ?.All children were followed up for one year,recording the annual bleeding rate(ABR),the annual target joint bleeding rate(ATJBR),Haemophilic Early Arthropathy Detection with Ultra Sound(HEAD-US),Hemophilia Joint Health Score(HJHS),and the factor ? for inhibitors.Statistical analysis was performed on the above five indexes,and P<0.05 was statistically significant.ResultsBleeding events occurred repeatedly in the on-demand group,and prophylaxis was more effective in improving bleeding than the on-demand treatment,and there were differences among different doses prophylaxis.The intermediate-dose group was superior to the low-dose group,and the standard-dose group was superior to the intermediate-dose group.The changes of target joint ultrasound score and the total score of HJHS in prophylaxis group were smaller than those in the on-demand treatment group,and there was no statistical significance in the changes of ultrasound score between the low-dose prophylaxis group and the intermediate-dose group(P>0.05).The change of HJHS total score in the intermediate-dose group was smaller than that in the low-dose group.The ultrasound score and HJHS score of the standard-dose group decreased compared with the baseline level,indicating an improvement trend(all P<0.05).None of the patients developed inhibitors during follow-up.Conclusionprophylaxis can obviously reduce the number of bleeding compared with on-demand treatment.However,for the improvement of joint structure and function,there are still differences between different doses of prophylaxis.Low-dose prophylaxis cannot prevent the progression of joint damage.Compared with low-dose,intermediate-dose prophylaxis can avoid progressive damage of joint structure and function.Standard-dose prophylaxis can significantly prevent or even improve the progression of hemophilia joint disease and protect joint function more effectively.Joint ultrasound score and HJHS score can reflect the situation of target joint timely and effectively.Long-term prophylactic infusion of clotting factor ? did not increase the incidence of inhibitor.