Biotech Values

[DewDiligence]

GTCB
This story of a woman with
hereditary antithrombin deficiency
was posted on the Yahoo board
this evening. It has a happy ending.

http://www.atiii.com/patientvoice.htm

>>
Taylor has HD
A PATIENT'S VOICE

I have known for a long time that I was antithrombin deficient. My first DVT occurred when I was eighteen and just about to start my sophomore year of college. Almost overnight, my left calf swelled and really hurt. My mother had developed a DVT after delivering me, so she recognized the signs and took me to a vascular surgeon.

Back then, gentle, painless sonograms used to confirm DVT were not around. Instead, doctors diagnosed DVT by injecting dye into a vein in your foot - a painful procedure. Doctors were also less familiar with blood protein deficiencies that can cause DVT -- like antithrombin and Factor V Leiden. Since I wasn't obese, on birth control, or injured, the doctors couldn't figure out why I got it. They thought it was a fluke. So, after standard heparin therapy, I went back to college on a 6-week coumadin regimen and promptly forgot about it.

But that DVT was not a fluke. Over the years I had several more DVT recurrences at the most inopportune moments. Just before final exams during my last year of law school, for instance, I got another one. From the symptoms, I knew that it was probably another DVT. I tried to tough it out and get through my exams before I called the doctor. With the bar exam a few months away, and a job to consider, I couldn't afford to delay my graduation. But sticking it out was wishful thinking. The pain was too much and I had to call the doctor. The next morning we met at the emergency room and a sonogram confirmed DVT. Although it was my second, seemingly inexplicable DVT, the doctors didn't initially suspect antithrombin deficiency, nor did anyone know I was at risk for developing a PE on standard heparin therapy alone.

A few nights later I got a PE and all I could do was press the hospital's "help" button by my bed. The night nurse peered into my room, didn't hear a peep (since I could barely breathe, much less speak), and assumed I was asleep. The next morning, the doctors confirmed that I had a large PE and immediately scheduled an operation to put in something called a "Greenfield filter." The Greenfield filter, new at the time, was used to filter out clots.

However, I developed extensive clotting. Today, this is a well-known side-effect of the device. So after being home for only two days, I was re-admitted to the hospital and treated with antithrombin and a
blood-clot dissolver.

After almost a month in the hospital, I graduated with the rest of my class, due to no small miracle. My law school professors were incredibly supportive and agreed to let me take my exams at home. Since I was just out of the hospital and couldn't get around very well, they arranged for a runner to ferry the exams to my door and back. They dropped everything to grade my exams immediately so I could meet the school's deadlines. It was like a baton race and, without their help, I would have never graduated on time.

A few years later on the eve of a big jury trial, I got another DVT when I had to stop taking coumadin for a non-elective surgery. Over the years, it was finally confirmed that I had the genetic condition called hereditary antithrombin deficiency, and that I will always have to take an anticoagulant, like coumadin, to prevent DVT.

After that third episode, my legs were never the same so I had to adjust my activity level. These events also settled any lingering doubt that I might be able to have children. This last DVT proved that as a person with antithrombin deficiency, I will get a DVT if I am not on coumadin. These were disappointing set-backs, to be sure, but I still had a good life.

This could be the end of my DVT story, except this is where it really begins.
In November of 1999, I met a terrific man and we started dating. He was OK with the fact that I couldn't have children, although he would have liked them, and we decided to get married.

On July 10, 2000, we had a small wedding and a great honeymoon. We were careful in our "family" planning, but I got pregnant, although I didn't know it at the time. When I had the first inkling I might be pregnant, I immediately stopped taking coumadin, since I knew it can cause birth defects, and promptly sought the advice of a hematologist.

My hematologist put me on Lovenox - low molecular weight heparin - and co-managed my pregnancy with a superb OB/GYN group specializing in high-risk pregnancies. The hematologist recommended Lovenox because it is safe in pregnancy, but by no means did anyone deem it a perfect solution. Heparin needs to bind with antithrombin to be effective. It is well-established that virtually all women experience a deficiency in antithrombotic blood proteins during pregnancy. So for me, being pregnant could only make my antithrombin deficiency worse.

Understandably, the doctors were concerned and took care to ensure I was fully aware of the risks of continuing the pregnancy. If the Lovenox didn't work -- and no one could say it would -- I was at a tremendous risk for developing life-threatening DVT's. Moreover, I was acutely aware the baby might have birth defects because I had taken coumadin for a few days during the first trimester, before I knew I was pregnant.

Thankfully, sonograms showed that the baby looked fine. Throughout the first several months of my pregnancy, I did fine, taking twice-daily shots of Lovenox and "natural" blood thinners like garlic, salmon, and vitamin E. But even with all these precautions, I got a DVT just as I was beginning my sixth month. I went into the hospital for a week and received heparin and antithrombin.

When I was released from the hospital, I had no choice but to follow the doctors' advice and resume coumadin. Clearly the Lovenox had not worked and coumadin was the only thing that would prevent another DVT. But coumadin still carried a risk of birth defects -- albeit a much smaller one. Resuming coumadin also meant the delivery had to be scheduled a good three weeks early to minimize the baby's exposure.

For the time being, the DVT situation was under control, but a bigger problem was yet to come: managing the delivery. The hospital had given me its very last bag of antithrombin during my last stay. In order to prevent another DVT, I had to be treated with antithrombin, both before and after the delivery. My hospital was completely out of it, and so were the hospitals nearby. As we would soon discover, there was a nationwide shortage of Bayer's antithrombin, which is made from human blood; Bayer confirmed the shortage would continue. No one, including the doctors, could do a thing about it.

The next few months were literally a roller coaster ride. It was hard not to panic as I worried about what might happen if I had the baby without antithrombin. Staying on coumadin for the delivery could cause excessive bleeding, not to mention that coumadin therapy makes it impossible to get an epidural. It was clear that I had to find antithrombin; giving up was not an option.

As the delivery date drew closer and closer still, we could not find any antithrombin, anywhere. Then we had an amazing turn of events. A law school friend learned of my plight and helped put me in touch with a doctor who knew about a company developing transgenic antithrombin. GTC Biotherapeutics (or "GTC") had developed an antithrombin product that -- unlike Bayer's -- did not depend on donated human blood for its production. We contacted GTC and they immediately agreed to donate recombinant antithrombin - still in development -- so long as the FDA would allow it.

With only days to spare before the delivery date, truly another miracle came. GTC received the FDA's approval to donate the drug on a compassionate use basis. The company had put everything in place so that if the approval came, they would be ready to send the antithrombin to my hospital. A few days later, on May 10, 2001, the doctors delivered a perfect little boy by C-section.

Today he is a beautiful and rambunctious healthy four-year old. How blessed I am to live every day with this little miracle. And how grateful I am that my story is just beginning.

Taylor
November 2005
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