Prevention of joint damage in hemophilic children with early prophylaxis

Radiological and orthopaedic outcome in severe and moderate haemophilia A a nd B patients undergoing long-term prophylactic treatment were prospectivel y investigated focusing on the age of onset of prophylaxis and the number o f joint bleedings prior to treatment. We report on 21 patients with severe and moderate haemophilia A and B receiving prophylactic treatment of betwee n 3.1 and 16.1 years duration. Three patient groups were evaluated accordin g to the age at onset of prophylaxis. In group I (n = 8) prophylactic treat ment was initiated in the first 2 years of life. Patients of group II (n = 6) received prophylaxis at the age of 3-6 years. Late-onset or secondary pr ophylactic treatment was started at the age of 6 years and above in 7 patie nts (group III). All patients received virus-inactivated F VIII or F IX con centrates at dosages of 30-40 IU, in some cases up to 50 IU/kg body weight i.v. three times per week for those with haemophilia A and twice per week f or those with haemophilia B. Elbow knee and ankle joints were investigated at 3-4 yearly intervals according to the radiological and orthopaedic score s recommended by the World Federation of Haemophilia (WFH). The total numbe r of joint bleedings before and after start of prophylaxis were recorded in all patients. In group 17 out of 8 patients had unaffected joints with con stant radiological and orthopaedic scores of zero or 1, after a median of 1 1.25 yea rs of prophylactic treatment. One patient in this group demonstrat ed mild radiological alterations (score 4). Patients of group II showed nei ther radiological nor orthopaedic alterations at study entry. Worsening joi nt scores could be detected despite ongoing prophylaxis after the 3-year in terval (median orthopaedic score 4, median radiological score 8). Treatment group III already showed considerable joint damage at study entry with a m edian radiological score of 11 (0-33) and a median orthopaedic score of 4 ( 0-11). Despite prophylactic treatment both, orthopaedic (median 8, range 2- 12) and radiological scores (median 19.5, range 2-47) deteriorated after 3 years. Prior to onset of prophylaxis no or only one joint bleeding occurred in treatment group I. In group II, a median of 6 joint bleeds (range 1-8) were reported before prophylaxis was started. Patients of group III usually experienced a med lan of more than 10 joint haemorrhages (range 6-10 or mo re). Under prophylactic treatment the number of joint bleedings decreased s ignificantly in groups II and III. However, radiological and orthopaedic sc ores increased as a sign of progressing osteoarthropathic alterations in pa tients reporting more than 6 joint haemorrhages before onset of prophylaxis whereas no joint alterations could be assessed in patients with no or only one joint bleeding episode prior to prophylaxis. Even a small number of jo int bleedings seems to ca use irreversible osteoarthropathic alterations le ading to haemophilic arthropathy. Once apparent, further progression of joi nt damage could not be arrested despite of prophylactic treatment (group II and III). In order to prevent haemophilic arthropathy, effective prophylax is should be started before or at least after the first joint bleeding in s evere haemophilia A and B.