clotting factor viii

ambermichelle · Apr 22, 2009

I am the mother of a factor VIII deficient hemophiliac son. What do you want to know?

Recombinant VIII products are newer, generally more expensive, and are synthetic rather than based on human blood products. Thus they are safe from HIV, hepatitis, and any virus not yet discovered that may someday get into the blood supply and plague humanity (as HIV did in the 80s).

I have not read the MASAC lately but I would hope that recombinant products are the standard of care, at least for anyone who was negative titer for the above viruses. Factor VIII can be given either as needed or prophyllactically several times a week to keep Factor VIII level up in the blood and avoid bleeding episodes, which can be life threatening or crippling. The products are amazingly expensive. Dosage is based on body weight and a dose for my petite son might be 2700 units, at a buck a unit. This is prescribed for several times a week. So maybe $30000 a month. We have always had good insurance that picked up the whole thing, but he is about to age out of parental insurance, and I dread it.

Human blood products are safe now also thanks to many processes to inactivate the known viruses. They have an excellent record for several decades of not transmitting viruses. But there is a theoretical risk that some of the virus may not be fully attenuated, or a new virus evolves that slips through the current very effective methods.

A fair number of hemophiliacs develop an inhibitor to Factor VIII which renders it useless. These unfortunate souls must switch to alternate products such as Factor VII products, that in our experience do not work well. Some, like my son, have success with immune tolerance which consists of daily high doses of Factor VIII for months until the inhibitor is overwhelmed, or until the insurance company stops paying. After a year of this, my son is back to responding well to Factor VIII.

(Soapbox rant, sorry) My son didn't ask to be born with this rare disease. We did not even know I was a carrier until he was diagnosed. He has had a very hard time and is my hero, because he has toughed it out and is about to graduate from college in accounting. But with employer based insurance and no universal health care option, if he does not somehow find a job in this rotten economy, he will not be able to afford his meds. No insurance company will give him a policy. The local county system cannot afford them either. In the past when we were on the "treat when needed" scheme, he had spontaneous subdural hematomas, an ab bleed as big as your fist, and a shoulder bleed as big as a football pad. I am afraid if he does not infuse regularly, bad things will happen.

ambermichelle · Apr 23, 2009

There is a National Hemophilia Foundation MASAC committee recommendation on recombinate vs plasma derived factor VIII. They say recombinate is safer but seem to still allow plasma..try Google, "MASAC Recommendation #187". You might ask a hematologist or a hemophilia nurse at one of the Hemophilia Treatment Centers, if you have access to one. Other than the odd emergency, hemophilia is usually handled by those who specialize in it--not every hematologist and certainly not every physician or nurse will be able to answer specific questions. Many have never encountered the disease in their practices.