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"Lucky" 13 left

Posted Aug 26 2008 12:34pm
Wow, it's just ticking down! I did -14 last night (yes, we're counting backwards now), and realized a certain symmetry... if you dig back, I did a rather lengthy post at 13 shots in, about forgetting to pick up my meds and having to hit the after-hours pharmacy. Anyway, I don't have much of anything to write with respect to treatment, side effects, etc. except that I've recently become aware of a dull steady headache and a mild fatigue. It's been going on for a couple of weeks, now that I stop to think about it. Could be treatment... could just be the change of seasons, to which I've always been pretty sensitive. *shrug*

So my last post set off a rather lengthy thread which turned toward some interesting news reported last week, and I figured I'd address it here, along with taking the opportunity to do a bit of "hemophilia 101". If you want a really comprehensive crash course in the various bleeding disorders, the National Hemophilia Foundation web site does a good job, particularly of explaining Hemophilia A (FVIII) , Hemophilia B (FIX) and VonWillebrand's Disease .

To give some perspective: Hemophilia A and B affect a combined total of approximately 20,000 Americans (roughly 1 in 5000). The NIH put forth the estimate several years ago that there may be as many as 2 million Americans affected by Von Willebrand's disease (a little less than 1%), but this number is hard to pin down, since VWD is poorly recognized or even known, and awareness is so low, that diagnoses are often missed.

For purposes of this discussion, I'll primarily be referring to Factor VIII deficiency, which I've been living with for all of my 34+ years, and thus understand best. There are actually 13 proteins in Human blood which work in conjunction (a sequence called the "clotting cascade" - see graphic below) to form a Fibrin clot. This is the mechanism that controls internal bleeding (typically into joint cavities or muscles); cuts and scrapes involve a different clotting process (scab formation, etc.), and generally affect hemophiliacs in a fashion similar to anybody else.

Sidebar tip: Wanna' annoy a hemophiliac? Just ask them if having hemophilia means they bleed to death if they get cut. It's nothing personal... we've just gotten tired of answering that question over and over.

Ok, so back to the "cascade": last week, the Baltimore Sun ran a story (which spread to other papers around the country) about how the US Military is treating combat trauma using Factor VII. Note from the graphic that FVII comes into the cascade from a different "direction" than the other clotting factors. This is a graphical representation of how FVII acts as a sort of supporting protein to the process, but behaves differently than the other proteins and is not integral to the cascade itself. Its unique characteristics and action pathway explain why doctors are exploring its use to treat combat injuries.

In hemophilia care, Factor VII is used to treat individuals who are specifically FVII deficient (very rare!) and Hemophilia A & B patients who have developed an inhibitor. Essentially, an inhibitor is a condition where the patient's immune system is activated against the factor injections, thereby rendering them useless to treat bleeding episodes and putting the patient in a VERY precarious position. Factor VII is administered as a substitute in this situation, and while it's not the most effective way to get to a clot, it works sufficiently well to prevent the patient from having to suffer through a bleeding episode without any treatment whatsoever.

Implications of Giving FVII to Troops

I have some seriously mixed feelings about this application of FVII. Let me be clear from the outset that I believe our troops should have the best medical attention available , regardless of my feelings about this poorly justified, poorly planned, poorly executed, and poorly managed adventure in Iraq staged by and for a bunch of greedy old jackasses who can't differentiate sending young men and women into peril from your average video game.

Administration of FVII is a very complicated issue, however. On one hand, there is a risk of causing stroke or other potentially fatal conditions when clotting factor is given to a non-hemophiliac patient. (The article covers this in some detail, so I won't elaborate further) Moreover, this product is very expensive (upwards of $6000 per dose) and not produced in massive quantity. Certainly, Novo Nordisk (the Danish pharmaceutical that produces Novo Seven) may be able to ramp up their production, but I worry about availability to the hemophilia community, that has an established (as opposed to an experimental) need for this product. In all fairness, Novo is getting clobbered in hemophilia treatment by a competitor's product, and may simply be looking for another market channel to distribute its goods... it seems to me to be drifting dangerously close to war profiteering, however.

My last concern is that this may be embraced as the standard of care and implemented in trauma centers throughout the US (and maybe the developed world), which would only amplify the problems mentioned above.

On the other hand (as it was pointed out to me by a close friend), this may be a very good thing for the hemophilia community, in that it will raise awareness of what our treatments cost, and more ERs and trauma centers will know how to treat a hemophiliac when he (or very rarely she) shows up in need of treatment. Also, given the new Congress and the promises of aggressive oversight, we might just see a meaningful dialog begin about how expensive clotting factor therapies are, and Congress might just figure out an effective way to curb the runaway profits of big pharma and ensure access to treatment (something we're all perpetually worried about)... at least in this one little area of medicine. Then again, the pharmaceutical lobby is one of the most powerful in Washington, so I'm skeptical.

So we can only sit back and wait for this to unfold.
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