Gene/Protein Disease Symptom Drug Enzyme Compound
Pivot Concepts:   Target Concepts:
Query: UMLS:C1832526 (PCC)
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In summary, while a great deal of information has accumulated concerning the properties and natural history of F VIII inhibitors, management remains frustrating and controversial. While bleeding episodes in those who are low responders can be treated with F VIII concentrates, treatment of bleeding in high responders is often much more difficult. Current therapeutic options include F VIII concentrates of human or porcine origin in high dosage, and PCC or APCC. The choice of treatment depends on the patient's current inhibitor concentration, the type and severity of bleeding, product availability, and the preference of the medical personnel involved. However, none of the available therapeutic modalities work as well as F VIII in a hemophiliac without an inhibitor. Perhaps more promising are the immune tolerance regimens that have been developed and are now being modified and fine tuned by a number of investigators. Such regimens have reportedly eradicated F VIII inhibitors in some hemophiliacs, and have converted others from high responders to low responders, in whom bleeding episodes can be effectively treated with conventional doses of F VIII. In contrast to the F VIII inhibitors developing in hemophiliacs, those developing in nonhemophiliacs can often be eradicated with corticosteroids or immunosuppressive drugs, either alone or in combination with F VIII. Not all respond to such approaches and serious hemorrhage may still occur. Treatment of bleeding episodes has included the use of human or porcine F VIII, APCC and, in two instances, DDAVP.
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PMID:Management of patients with factor VIII inhibitors. 298 Feb 69