Long-term results of the treatment of children with factor VIII inhibitors with prothrombin-complex concentrates.

Twelve boys with haemophilia and factor VIII inhibitors have received initial treatment for each episode of bleeding with prothrombin-complex concentrate. The follow-up period for these boys has ranged from two months to 14 years, and a total of 732 bleeding episodes were reviewed. Each boy underwent a full clinical and radiological assessment of knee-joint function, and this was compared with knee-joint function in 29 patients with severe haemophilia but without factor VIII antibodies. A change of treatment from prothrombin-complex concentrate to some other form of therapy was considered to be required in only 4% of the bleeding episodes. Of 10 severe bleeding episodes, five episodes clearly were controlled with prothrombin-complex concentrate, but five episodes necessitated alternative treatment. Four of the 12 families considered prothrombin-complex concentrate as relatively ineffective. All boys who were older than 10 years of age showed grade-1 or grade-2 impairment of knee-joint mobility, as well as radiological evidence of moderate-to-severe knee-joint changes. In the group of haemophiliac patients who were older than 10 years of age and did not have factor VIII antibodies, only 10 of the 58 knee-joints that were examined showed grade-1 or grade-2 functional impairment. Although prothrombin-complex concentrates were effective in the control of the majority of minor bleeding episodes, they were effective in only 50% of severe episodes of bleeding and did not prevent the early development of haemophilic arthropathy.