Efficacy and Safety of Third-Generation Full Length Recombinant Human Coagulation Factor VIII in the Standard Prophylactic Treatment of Childhood Patients with Moderate-to-Severe Hemophilia A: A Single Center Experience

Objective To evaluate the number of annual bleeding and annual joint bleeding and safety of third-generation full length recombinant human coagulation factor Ⅷ (ADVATE) in the standard prophylactic treatment of childhood patients with moderate-to-severe hemophilia A. Methods Fifteen children aged from 5 to 18 years with moderate-to-severe hemophilia A were enrolled in the one center, prospective, nonrandom, self-control study from Nov 01,2014 to Nov 30,2016. Children were treated with one-year long-course infusion of ADVATE with a dose of 20 ∼ 40 IU/kg twice-weekly or three times weekly. The numbers of annual bleeding and annual joint bleeding after the prophylaxis treatment were compared with that on-demand. Moreover, any adverse effect and serious adverse reaction related to ADVATE usage were documented during the study. The FⅧ inhibitor titer were measured with modified Nijmegen method before and after the prophylaxis every 3 to 6 months. Results The FⅧ:C for these 15 boys, median age 15, was less than 2.2%, with 8 patient severe type. The number of annual bleeding was reduced from 27.6 on-demand treatment to 2.6 after the prophylaxis treatment( P < 0.001), and the number of annual joint bleeding was also reduced from 11.1 to 0.9( P < 0.001). Seven patients had zero annual joint bleeding and the mean annual bleeding for mild, moderate and severe degree was decreased significantly with zero times for severe degree bleeding after the standard prophylactic treatment. No adverse effect or serious adverse reaction related to ADVATE was recorded during one-year period treatment and FⅧ inhibitor detection was negative. Conclusions Standard prophylactic treatment with ADVATE can effectively decrease the annual bleeding and annual joint bleeding in children with moderate-to-severe hemophilia A. This therapy is safe with FⅧ inhibitor test negative.