What is the prognosis of factor IX deficiency (FIX) (hemophilia B)?

Updated: Mar 09, 2021
  • Author: Robert A Schwartz, MD, MPH; Chief Editor: Srikanth Nagalla, MD, MS, FACP  more...
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Answer

Prognosis depends on the types of complications that develop, as well as the type of product replacement available when the patient started undergoing care. Currently, younger patients with hemophilia who receive recombinant products do much better than patients who received the older products. Gene therapy for these disorders is currently under evaluation.

Early and complete genetic testing of all persons newly diagnosed with hemophilia is key to anticipating and preventing serious complications.

Preventing or suppressing the anamnestic rise of FIX inhibitors in patients with severe FIX deficiencies may be feasible with the use of monoclonal antibodies, which target T-cell response to antigenic stimulation. The blockade of CTLA4 and CD28-B7 interactions with T cells is shown to have implications for successfully preventing destructive T-cell responses in autoimmune disease.


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