Treatment of children with haemophilia in Europe: a survey of 20 centres in 16 countries

Abstract

A survey was made of the current status of treatment of haemophilic boys at 20 centres in 16 European countries and includes approximately 1500 of the estimated 6500 haemophiliacs in the participating countries. Many mild haemophiliacs are not seen, or seen infrequently, at haemophilia centres and this requires study. Nine of 18 centres provide continuous prophylaxis to 80-100% of their patients, five centres provide it to 55-80% and the remaining four centres to 15-40% of the boys. The median dose given was 6240 U kg-1 year-1 (range 3120-7800). Four centres administered only recombinant concentrates to children with severe haemophilia A, while seven centres administered recombinant concentrates to 75-90% and the remaining centres to less than 50% of the boys (two centres < 10%). When asked for the choice of concentrate for a newly diagnosed boy with severe haemophilia A, all but one centre preferred recombinant concentrate. Most boys below 6 years received concentrates via a peripheral vein but three centres preferred a central venous line for 80-100% of the boys. Thirteen of 18 centres applied home treatment to 84-100% of the boys and the remaining five centres to 57-77% of the boys.

The effects of postponing prophylactic treatment on long-term outcome in patients with severe hemophilia

“…No. of joint bleeds until prophylaxis 0.5 (0-1) 11 (7)(8)(9)(10)(11)(12)(13) 32 ( In conclusion, most patients have their first joint bleed after the age of 2 years. Patients who start prophylaxis soon after the first joint bleed show little arthropathy in adulthood.…”

“…Although prophylaxis was postponed even longer for patients in the last category (Ն 45 joint bleeds before prophylaxis), both their Pettersson scores and clinical scores were comparable to those of patients who started prophylaxis after 15 to 44 joint bleeds. The median Pettersson score for all patients who had at least 15 joint bleeds before prophylaxis was 11 points (IQR [3][4][5][6][7][8][9][10][11][12][13][14][15][16][17][18][19][20]. Analysis of the adjusted effect of the number of joint bleeds before starting prophylaxis on Pettersson scores after 19 years of follow-up showed a 7% higher Pettersson score per 10 extra joint bleeds before prophylaxis (RI 1.07, CI 1.00-1.15, P .14).…”

“…Some centers advocate starting prophylaxis before the first joint bleed (ie, around the age of 1 year), [7][8][9] while others wait until one or more joint bleeds have occurred. 10,11 Since its introduction in 1968, prophylactic treatment for severe hemophilia in The Netherlands has been started at increasingly younger ages but generally after the occurrence of the first joint bleed. 12 Currently, no prospective studies have been performed on when to start prophylaxis.…”

A modeling approach to evaluate long-term outcome of prophylactic and on demand treatment strategies for severe hemophilia A

“…However, the use of clotting factor is very expensive and this may be one of the main factors why prophylaxis is not adopted as standard treatment in all Western countries. 5 For example, the annual consumption for a young adult on prophylaxis in the Netherlands would be 105,000 IU. 6 In Sweden, higher dosages are used, and the average consumption would be 280,000 IU/year.…”

Hemofilia A dengan Komplikasi Epilepsi Pasca Perdarahan Intrakranial

“…[1][2][3][4] Meskipun demikian sekitar 30% kelainan ini akibat mutasi spontan pada ujung lengan panjang kromosom x. 2,3,[5][6][7] Perdarahan intrakranial merupakan salah satu penyebab kematian pasien hemofilia. Angka kejadian perdarahan intrakranial pada pasien hemofilia adalah 2,2%-13,8% dengan angka kematian sekitar 25%-30%.…”