Recombinant Therapy for Hemophilia A Could Potentially Create Inhibitors


Patients receiving Recombinant Factor VIII had an 87% higher chance of developing inhibitors compared with patients receiving plasma-derived therapy.

A recent study found that patients receiving recombinant therapy for hemophilia A were able to create antibodies that inhibited the treatment at twice the rate of patients receiving treatments derived from human plasma.

Recombinant Factor VII, which is derived from a hamster cell line, is associated with an increased risk of developing inhibitors that can make the treatment ineffective compared with Factor VII, which is derived from human plasma, according to the study published in the New England Journal of Medicine.

"Families will want to have a discussion with their physicians about how this study might impact the treatment options," said researcher Mindy Simpson, MD.

Currently, Recombinant Factor VII is the preferred treatment due to a large amount of patient deaths in the 1980s due to blood supply tainted with HIV and hepatitis C virus.

“Patients and families have been afraid of plasma-derived products even though they have been safe for decades," Dr Simpson said

The Survey of Inhibitors in Plasma-Products Exposed Toddlers (SIPPET) study included 251 previously untreated children, and compared both of the first-line treatments for hemophilia A. Researchers found that approximately 30% of patients receiving recombinant therapy developed inhibitors within the first 50 treatments.

The study concluded inhibitors that appear in high volumes per unit of blood render factor VIII replacement therapies useless, and patients must then switch to treatments that are more expensive and potentially less effective.

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