Commercially produced factor concentrates such as “Advate”, a recombinant Factor VIII produced by Baxter International, come as a white powder in a vial which must be mixed with sterile water prior to intravenous injection. (Photo credit: Wikipedia)
Hemophilia is an uncommon genetic condition that results in difficult-to-control bleeding, usually in boys. The underlying disorder is a deficiency of certain blood factors that are required to make normal clots, called Factor VIII and Factor IX among others. Accepted treatment includes giving these factors to these patients. This is highly effective treatment unless the patient begins to form antibodies against the synthetic factors, which most of these boys do eventually. They tend to require higher and higher dosages over time in order to prevent bleeding episodes, and some become treatment-resistant.
A new product has hit the market that may change all of that. Called FEIBA NF (Baxter Pharmaceuticals), the medicine bypasses the need for these escalating doses of factors. This study looked at giving FEIBA NF as a maintenance dose as opposed to using it only when bleeding occurs. The results were dramatic. Bleeding episodes were reduced by nearly 3/4 with no increase in adverse effects.
Using modern treatments, a condition that had been uniformly fatal has become a controllable chronic disorder. This is a powerful tool to have in the toolbox!
Antunes, S. V., Tangada, S., Stasyshyn, O., Mamonov, V., Phillips, J., Guzman-Becerra, N., Grigorian, A., Ewenstein, B. and Wong, W.-Y. (2014), Randomized comparison of prophylaxis and on-demand regimens with FEIBA NF in the treatment of haemophilia A and B with inhibitors. Haemophilia, 20: 65–72. doi: 10.1111/hae.12246