TREATMENT OF ACUTE BLEEDS WITH RECOMBINANT ACTIVATED FACTOR-VII DURING IMMUNE TOLERANCE THERAPY

In our clinic, five patients with haemophilia A and one patient with h aemophilia B and inhibitors have been treated with immune tolerance in duction (ITI) since 1995. Bleeding symptoms during this period have be en treated with recombinant activated factor VII (rFVIIa; NovoSeven, N ovo Nordisk, Bagsvaerd, Denmark). Four of the six patients did not nee d rFVIIa during ITI other than for port-a-cath insertions, but two hav e been treated intensively because of repeated bleeding problems. The first of these developed inhibitors at the age of 2 years after 11 day s of exposure to factor VIII (FVIII). He was treated for 40 bleeding e pisodes before ITI started, and during ITI he was treated another 24 t imes, including eight treatments for joint bleeds. Treatment was effec tive for the different types of bleeding episode. However, in spite of repeated treatment for these joint bleeds, he developed two target jo ints with synovitis (right knee and left elbow). The synovitis only sh owed signs of regression when inhibitor levels were reduced due to the ITI regimen. The second patient, now 5 years old, has severe haemophi lia B. He developed inhibitors and anaphylaxia having received prophyl actic treatment from the age of 1 year. He has now received In with 12 0 units/kg body weight per day of FIX for 68 weeks. In the event of tr auma and bleeding he is treated promptly with rFVIIa by his parents at home. Treatment is started with 160-180 mu g/kg body weight and, if n eeded, another dose of 90 mu g/kg is given after 3 h. During 1996, 35 bleeds or traumas were treated. The total amount of rFVIIa administere d to this child was 211.2 mg. All but one joint bleed and all muscle b leeds needed more than one injection. The need for another injection i s judged by the parents and the child from clinical signs, such as pai n and swelling. Neither of these two boys have shown any signs of thro mbosis or disseminated intravascular coagulation. In summary, rFVIIa i s a well tolerated and effective therapy for acute bleeding episodes d uring ITI. Dosing and intervals can be the same as for patients not on ITI therapy. Early intervention at home can minimize the risk of syno vitis. Blood Coag Fibrinol 9 (suppl 1):S143-S146 (C) 1998 Lippincott-R aven Publishers.