Abstract
Objective: To assess the role of early prophylaxis with recombinant activated factor VII (rFVIIa) in young haemophiliacs with inhibitors and to determine whether it can reduce bleeding episodes and prevent joint damage. Patients and Methods: Ten severe haemophiliacs, less of three years old, with up to four joint bleeds and inhibitors to FVIII who started early prophylaxis with rFVIIa, were included. A number of haemorrhages/year/patient and haemarthroses/ year/patient were compared before the start of prophylaxis, which include both the time before (initial period) and after inhibitor diagnosis (inhibitor period), with those during prophylaxis (prophylaxis period). Results: The mean time of inhibitor diagnosis was 15.6 months (range: 2.3-34.1). The mean time between inhibitor diagnosis and the start of prophylaxis was 7.1 months (range: 0-23.2), shorter than the time of rFVIIa prophylaxis (mean: 10.3 months; range: 4.1-32.0). Bleeding episodes for the three time periods were 45, 36 and 17, respectively, or 0.29 and 0.51 haemorrhages/year/patient in the two periods prior to prophylaxis compared to 0.17 during prophylaxis. Total haemarthroses for the three-time periods were 7, 5 and 5, respectively. The haemarthroses/year/patient in the pre-prophylaxis period were 0.032 and 0.070, compared to0.049 inthe prophylaxis period. rFVIIa schedules were either 90 μg/kg three times weekly or 90 μg/kg daily. Conclusions: Early prophylaxis with rFVIIa may be efficacious in young haemophiliacs with inhibitors, reducing joint bleeds. After the risky period of inhibitor presence, they are able to continue rFVIII prophylaxis with success assured to prevent arthropathy.
Highlights
The benefits of early prophylaxis over on-demand therapy in severe haemophilic children that have not acquired antibodies to clotting factor replacement therapy have been established [1]
Ten severe haemophilia A patients, who were less than three years old and had developed high-responding inhibitors after treatment with recombinant Factor VIII (FVIII) were included in the study
Compared with previous on-demand therapy, Konkle et al found the frequency of bleeds was significantly (p < 0.0001) reduced by 45% for 90 μg/kg rFVIIa and 59% for 270 μg/kg rFVIIa during prophylaxis in 22 patients who had high requirements for on-demand therapy and were given prophylactic treatment daily for three months [12]
Summary
The benefits of early prophylaxis over on-demand therapy in severe haemophilic children that have not acquired antibodies (inhibitors) to clotting factor replacement therapy have been established [1]. Prophylaxis acts principally to minimize joint bleeds and the subsequent OPEN ACCESS. López-Fernández et al / Health 5 (2013) 1151-1157 development of arthropathies and other life-threatening bleeding events, such as intracranial haemorrhage. Prophylaxis is widely accepted as the preferred mode of therapy for children with severe haemophilia. Paediatric patients with antibodies that inhibit clotting factor replacement therapy have a high risk for recurrent joint bleeds and subsequent haemophilic arthropathy [2]. Several studies on the orthopaedic status of patients with inhibitors found more severe arthropathy even in younger patients [3,4]
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Free) 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a call
