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To correlate the HLA type and genetic defect with hemophilia A.
One of the most serious complications of treatment in patients with hemophilia A or hemophilia B is the development of an inhibitor, which is an antibody that neutralizes the factor VIII or IX coagulant activity. Up to one fourth of patients with severe hemophilia A develop an inhibitor but at present it is not possible to predict which patients will develop such antibody. The ability to predict an inhibitor development at an individual level would greatly improve therapeutic approach to this serious problem
Observational Model: Case-Only, Time Perspective: Prospective
The Univeristy of Texas Health Science Center at Houston
The University of Texas Health Science Center, Houston
Published on BioPortfolio: 2014-08-26T22:50:33-0400
To identify the causative mutations in previously untreated patients with hemophilia A enrolled in the ReFacto® clinical safety and efficacy study CTN 93-R833-0XX/C9741-28, using two esta...
To collect and analyze data on females with hemophilia so as to better define the difference between the study population and the male population with hemophilia.
To determine the risk factors associated with inhibitor formation in hemophilia A and to study the mechanism of tolerance in the murine hemophilia A model.
To collect and analyze data on female carriers of severe and moderate hemophilia A and B.
To assess efficacy and safety of BeneFix® for prophylaxis in "Short-term" therapy and on demand therapy for all bleeding episodes of subjects with hemophilia B.
Hemophilia is the most common inherited coagulation disorder, and approximately one-half of patients are diagnosed as newborns. For prenatal diagnosis of hemophilia A, genetic tests are performed usin...
Hemophilia is an X-linked inherited bleeding disorder consisting of two classifications, hemophilia A and hemophilia B, depending on the underlying mutation. Although the disease is currently treatabl...
Inhibitor development is one of the major problems in hemophilia patients. Whereas the inhibitor incidence in hemophilia A is estimated to be as high as 25-30%, it appears to be less frequent in hemop...
Pre-clinical testing of new therapeutic strategies in relevant animal models is an essential part of drug development. The choice of animal models of disease that are used in these studies is driven b...
The prophylactic treatment of hemophilia involves the replacement of factor VIII or IX in affected persons. A new approach involves the depletion of the clot inhibitor antithrombin.
A deficiency of blood coagulation factor IX inherited as an X-linked disorder. (Also known as Christmas Disease, after the first patient studied in detail, not the holy day.) Historical and clinical features resemble those in classic hemophilia (HEMOPHILIA A), but patients present with fewer symptoms. Severity of bleeding is usually similar in members of a single family. Many patients are asymptomatic until the hemostatic system is stressed by surgery or trauma. Treatment is similar to that for hemophilia A. (From Cecil Textbook of Medicine, 19th ed, p1008)
The classic hemophilia resulting from a deficiency of factor VIII. It is an inherited disorder of blood coagulation characterized by a permanent tendency to hemorrhage.
A hereditary deficiency of blood coagulation factor XI (also known as plasma thromboplastin antecedent or PTA or antihemophilic factor C) resulting in a systemic blood-clotting defect called hemophilia C or Rosenthal's syndrome, that may resemble classical hemophilia.
Bleeding into the joints. It may arise from trauma or spontaneously in patients with hemophilia.
Stable blood coagulation factor involved in the intrinsic pathway. The activated form XIa activates factor IX to IXa. Deficiency of factor XI is often called hemophilia C.
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