Desmopressin increases endogenous factor VIII levels in hemophilia A. Large inter-individual variation in the response to desmopressin is observed. Patients with a lower baseline factor VIII activity tend to show a reduced response, therefore, desmopressin is less frequently used in moderate hemophilia A patients (baseline factor VIII activity 1-5 international units/deciliter), even though fac...

During a 4-year multicenter cooperative study of acquired factor VIII inhibitors in persons with hemophilia A. new inhibitors were detected in 31 of 1 .306 patients who entered the study without an inhibitor or the history of an inhibitor. The incidence of new inhibitors was eight per 1 .000 patient-years of observation. The factor Vlll:C level before inhibitor development was 0.03 U/mi in 29 m...

BACKGROUND BAY 81-8973 is a new full-length human recombinant factor VIII product manufactured with technologies to improve consistency in glycosylation and expression to optimize clinical performance. OBJECTIVES To demonstrate superiority of prophylaxis vs. on demand therapy with BAY 81-8973 in patients with severe hemophilia A. PATIENTS/METHODS In this multinational,randomized, open-label...

The pathogenesis of inhibitory antibodies has been the focus of major scientific interest over the last decades, and several studies on underlying immune mechanisms and risk factors for formation of these antibodies have been performed with the aim of improving the ability to both predict and prevent their appearance. It seems clear that the decisive factors for the immune response to the defic...

The objectives of this article were to study the reported prevalence of haemophilia B (HB) on a country-by-country basis and to analyse whether the prevalence of HB varied by national economy. The prevalence of HB is the proportion of diagnosed, reported cases of HB in a population at a specific point of time. We collected data on the HB prevalence for 105 countries from the World Federation of...

Chediak et al. have reported that the titer of procoagulant antihemophilic factor (AHF:C; factor VIII:C) was significantly lower in obligate carriers of classic hemophilia who were daughters of affected men (paternal carriers) than in those whose fathers were normal by history (maternal carriers). In contrast, among 113 obligate carriers of hemophilia, no significant difference in procoagulant ...

INTRODUCTION One of the recent advances in assessing outcomes of medical care is the inclusion of the patient perspective. The term patient-reported outcome (PRO) is used to reflect the patient perceptions of disease and its consequences as well as of treatment and health-care provision. The development of PRO measures has advanced rapidly, and implementation in clinical research and practice i...

Freeze dried cryoprecipitate was used in the treatment of 14 patients with haemophilia A. The in vivo recovery was 91.2% which is comparable to that reported from other parts of Europe. The product was efficacious and no adverse effects were reported. Freeze dried cryoprecipitate is the high yield product of a low technology process and as such may be of value in reducing any possible shortfall...

The Pan-Thames Haemophilia Consortium was established to provide coherent regional planning of haemophilia services. The Expert Advisory Group is a clinical reference point for the consortium and was established to consider specifically elective high cost treatment programmes. An audit of the first year of consortium activity demonstrated that patients receiving high cost therapy represented 1....

The paediatric care of children with haemophilia in developed countries should focus on the health of the child, not on the disorder. Gene therapy offers the hope of an ultimate 'cure' for the disorder, but until this is a viable proposition, patients should be given more control over their treatment, and the focus should be on 'self-monitored and self-adjusted' prophylaxis. New instruments for...