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29 boys (4-18 years old) with severe haemophilia A were given prophylactic infusions of AHF concentrate (human fraction I-0) for 2 to 13 years in an attempt to change the haemophilia from a severe to a moderate form and thereby prevent arthropathy and severe bleeding episodes. The sizes of the doses and the intervals at which the doses were given were titrated by AHF survival studies. As a rule, the patients received AHF in amounts sufficient to raise the AHF level to 30-45% at 5-12 day intervals. In about 50% of the infusions the AHF content was not below 1% before the next infusion. During such prophylaxis all patients except one have been in a good general condition. They have had bleeding episodes, which have, however, been much less severe and less frequent. The children have been able to live an almost normal life. The number and duration of stays in hospital have been markedly reduced. 17 of the patients had only minor or no joint defects before the start of the treatment. In this group the joint function was identical with that found in moderate haemophilia in the same age groups. Two patients developed anticoagulants. No other side effects were seen. The prophylactic regimen in Sweden thus reduced severe haemophilia to moderate.

The effect of prophylactic treatment of severe haemophilia A and B was evaluated with a recently described radiologic score system for assessing the severity of arthropathy. The severity of the arthropathy was assessed in 50 patients with severe haemophilia receiving prophylaxis with factor VIII or IX (25--30 units factor VIII or IX/kg bodyweight at 4--7 day intervals), and in 9 in whom the condition was complicated by inhibitors and who were thus not receiving prophylaxis. The score values found in a recent investigation of the natural course of haemophilia served as controls in the evaluation of the effect of the prophylaxis. In the group with severe haemophilia receiving prophylaxis changes appeared only exceptionally in joints not affected already before the prophylaxis, and the course of the arthropathy and its severity according to age did not vary to any noteworthy extent from that in the control group with moderate haemophilia. In the cases complicated by inhibitors the joints were most often worse than those in the control group with severe haemophilia. It is concluded that prophylaxis has a considerable effect on the course of the arthropathy and the prophylaxis should be instituted at an early stage of the disease, when the joints are still unaffected.

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