Screening of 43 healthy Danish haemophiliacs revealed a significantly lower helper/suppressor (H/S) ratio than in controls. 8 of the haemophiliacs had an H/S ratio less than or equal to 1.0. A significant negative correlation occurred between the total lifetime factor VIII treatment and the H/S ratio. However, high-dose factor VIII treatment given to patients with antibodies against factor VIII was not associated with immunological abnormalities. Children had a significantly higher H/S ratio than the adult haemophiliacs. Patients exclusively treated with Danish cryoprecipitate during the last year had a significantly higher H/S ratio than patients receiving preparations from other sources. This difference might, however, be explained by lower age and lower total lifetime dose in the group receiving Danish preparations. Haemophiliacs treated with American preparations did not differ immunologically from those treated with preparations of other origin. Total serum IgG was increased in 23% of the patients. This parameter was negatively correlated with the H/S ratio. The possible relation of the observed immunological alterations among otherwise healthy haemophiliacs to the acquired immune deficiency syndrome warrants further attention.
The prophylactic treatment of bleeding in von Willebrand disease (vWD) has a long history in Sweden, having been first initiated in the late 1950s. The clinical experience of the prophylaxis of vWD in Sweden is described in the current review of a retrospective study of 37 patients from three haemophilia centres receiving prophylaxis for vWD. Prophylaxis with a plasma concentrate (fraction I-0 or Factor VIII) effectively reduced the median number of bleeds per year, prevented joint arthropathy in those who started prophylaxis at a young age, and improved the quality of life of all patients with vWD, particularly those with type 3 disease. This study demonstrates that the long-term prophylactic treatment with von Willebrand Factor (vWF)/FVIII concentrate (Haemate-P, ZLB Behring) in vWD is warranted in the majority of patients with type 3 disease.
Canadian Blood Services' disposition reports suggested considerable variation in cryoprecipitate use and prompted this national audit.
Thirty-one institutions were invited to participate in a 2-month audit. Patient information and relevant laboratory and transfusion data were collected. Cryoprecipitate transfusions were categorized as appropriate if a fibrinogen level (taken 6 hr before/after transfusion) was not more than 1.0 g per L and inappropriate if the pretransfusion fibrinogen level was more than 1.0 g per L and posttransfusion fibrinogen level was more than 1.0 g per L or not performed. Appropriateness was categorized as undetermined if the pretransfusion fibrinogen level was not performed and the posttransfusion fibrinogen level was more than 1.0 g per L or not performed.
Overall, 25 of 31 invited hospitals agreed to participate. A total of 4370 units of cryoprecipitate were transfused in 603 events to 453 patients representing 62 percent of cryoprecipitate issued to hospitals during the time period. Comparison of the number of units of cryoprecipitate per 100 units of red blood cells (RBCs) transfused by each institution showed significant variation in practice (mean, 9 per 100 RBCs; range, 2 to 27 units). The single most common indication for cryoprecipitate was cardiac surgery (45.4% of events). Overall, 24 percent of cryoprecipitate transfusions were considered to be appropriate (pretransfusion fibrinogen level
In Canada, care for individuals with inherited bleeding disorders, including Von Willebrand disease, is provided by 26 tertiary care multidisciplinary Inherited Bleeding Disorders clinics geographically spread across the country. The Association of Hemophilia Clinic Directors of Canada, the Canadian Association of Nurses in Hemophilia Care, the Canadian Physiotherapists in Hemophilia Care, the Canadian Social Workers in Hemophilia Care, and the Canadian Hemophilia Society all collaborate to provide optimal management for patients with inherited bleeding disorders. The standards of care for these patients were explicitly laid out in a 2007 document published by the Canadian Hemophilia Standards Group (with representation from all of the groups just mentioned) entitled Canadian Comprehensive Care Standards for Hemophilia and Other Inherited Bleeding Disorders. Separate Canadian guidelines for the management of patients with Hemophilia and Von Willebrand disease also exist, focused on diagnosis, comprehensive care, assessment, and treatment.
Both Sweden and the Netherlands have a long experience with primary prophylaxis in children with severe haemophilia. In these countries it has been offered to all children for the last 3-4 decades. In Sweden prophylaxis is generally started at an earlier age with a higher dosage and frequency than in the Netherlands. Patients in the Netherlands receive a more individually tailored regimen, with prophylaxis now started after the first one or two joint bleeds and dosages are increased when breakthrough bleeds occur. The current study evaluated the effect prophylaxis on long-term outcomes and the consumption of clotting factor concentrates in Dutch and Swedish cohorts. Our results show that the orthopaedic outcome in the oldest groups of patients from Sweden and the Dutch cohorts were comparable, although the Swedish patients used twice as much clotting products per year. In the youngest patients, joint status is very good and further follow-up is necessary to demonstrate the benefits of either strategy. In conclusion, more individually tailored regimens aimed at preventing bleeding prevent joint damage in children with severe haemophilia, while clotting factor consumption is about half of that in previously described regimens.
OBJECTIVES: To review long-term prophylactic factor treatment in young patients with severe haemophilia A and B, focusing on the orthopaedic and radiological outcome. DESIGN: We received 34 patients with severe haemophilia A (n = 29) and B (n = 5), aged 7-22 years. Age at start of treatment was 1-4.5 years. Dosages of factor concentrate (F VIII and F IX, respectively) were 25-40 IU/kg body weight, three times a week for haemophilia A and twice a week for haemophilia B. The patients had been checked annually over a 5-year period (1990-95). Orthopaedic and radiological joint scores were evaluated according to recommendations by the World Federation of Haemophilia. SETTING: All results were obtained at the Department for Coagulation Disorders, University of Lund, Malm? University Hospital, Malm?, Sweden. RESULTS: Orthopaedic and radiological joint scores were found to have remained unchanged during follow-up in almost all patients and to be still zero (i.e. no unaffected joints) in 79% (n = 27) of the patients. CONCLUSION: There is a growing international consensus haemophilic arthropathy can be prevented by administering early high-dose prophylaxis. The results of the present investigation strongly support this opinion.