Hemophilia B is a rare X-linked recessive disorder with plasma factor IX (FIX) deficiency. 1-3% of patients treated with exogenous FIX-containing products develop inhibitors (i.e. polyclonal high affinity immunoglobulins) that neutralize the procoagulant activity of a specific coagulation factor. Although the incidence of inhibitors in hemophilia B patients is low, most are "high titer" and fre...

We gave danazol (600 mg/day orally for 14 days) to eight adults with mild or moderate hemophilia A, one with severe hemophilia A, and one with moderate hemophilia B. In the patient with severe hemophilia A, the levels of factor VIII two to four days after an infusion of factor VIII concentrate were higher than expected, suggesting a prolonged half-life. In one patient with mild hemophilia A, a ...

• Objective: To determine causes of death and mortality rates in patients with hemophilia over a period of 20 years, to assess changes in mortality, and to distinguish between hemophilia-related death and recent death induced by viral infections. • Design: Cohort study of 919 patients followed from January 1986 to June 1992. Results were compared with outcomes of previous follow-up from 1973 to...

The majority of cases of human hemophilia B are the result of missense mutations in the coagulation factor IX gene and defective circulating factor IX is detectable in most patients. The available mouse factor IX knockout models of hemophilia B (FIXKO mouse) reproduce the bleeding phenotype of human hemophilia B, but because the models produce no factor IX they fail to reproduce the dominant hu...

Hemophilia is a monogenic mutational disease affecting coagulation factor VIII or IX genes. The palliative treatment of choice based on the use safe and effective recombinant clotting factors. Advanced therapies will be curative, ensuring stable durable concentrations defective circulating factor. Results have so far been encouraging in terms levels times expression using mainly adeno-associate...

BACKGROUND Gene therapy could prevent bleeding in patients with hemophilia A, but might induce antibodies that block factor VIII (FVIII) function. OBJECTIVES To test the efficacy of gene therapy in the newborn period for preventing a response to human FVIII (hFVIII) because of immaturity of the immune system. METHODS Varying doses of a retroviral vector (RV) expressing a B domain-deleted hF...

Hemophilia A and B patients seen at nine US regional treatment centers were tested for serologic markers of hepatitis B virus (HBV), hepatitis C virus (HCV), and hepatitis delta virus (HDV) during 1987 and 1988. Because human immunodeficiency virus (HIV) infection, a potentially confounding variable, was present in 53% of the group, the population was divided by HIV status for analysis purposes...

Hemophilia B is an X-linked genetic deficiency of coagulation factor IX (FIX) activity associated with recurrent deep tissue and joint bleeding that may lead to long-term disability. FIX replacement therapy using plasma-derived protein or recombinant protein has significantly reduced bleeding and disability from hemophilia B, particularly when used in a prophylactic fashion. Although modern fac...

Hemophilic pseudotumors are rare but dangerous complications of Hemophilia. We hereby report a 3-year-old boy with Hemophilia B, presenting with nasal pseudotumor, showing favorable response to radiotherapy after unsuccessful treatment with factor IX replacement therapy. The diagnosis and treatment of this rare condition is also reviewed.

background: hemophilia b is a bleeding disorder with a recessive x-linked inheritance pattern, in which the infected individuals have low levels of factor ix in their plasma. affected individuals may have bleeding episodes after trauma or spontaneously considering the plasma level of factor ix. in order to prevent these episodes and to control bleeding, they should use coagulation factor concen...