NIH - The Rest of the Story

When Jo and I went to the NIH 2 weeks ago, there was actually a fair amount of concern stirred up when we talked about what has been going on the past year. Everybody was extremely concerned about the blood clot Jo had in her head last March shortly after her 2nd hip replacement. A blood clot in your head is very serious. It could lead to a stroke and irreversible brain damage. What surprised us a bit was that it seemed like ancient history to us.

Well, a blood clot is often an indicator used to by the NIH to make a decision to have a bone marrow transplant done, especially in a case like Jo’s where she has a perfect match donor. A bone marrow transplant is extremely difficult and has its own set of risks and jumping to transplant is never a decision made lightly. In Jo’s case, there is enough data to blame the clot on, that we have decided that we can continue down the path we are currently on and keep the bone marrow transplant as a backup plan.

Jo’s treatment for Aplastic Anemia is considered a tremendous success. Her bone marrow is producing good amounts of blood and most importantly, she does not require any blood transfusions. However, Jo also has the PNH problem where her red blood cells have a tendency to burst creating a bunch of cell garbage in her blood that puts her at high risk of clots. For that reason, she is on blood thinners at all times.

Blood thinners create their own problems. Jo has had to have several major surgeries and she will require a few more. When you have surgery, you cannot have thin blood. About 4 to 5 days before any surgery Jo stops taking Coumadin, her primary blood thinner. Coumadin stays in your system about 4 to 5 days, so she has to stop it in time for it to be completely out of her system before surgery. During that 5-day stretch, she gives herself shots of Lovonox, which only stays in her system about 12 hours. As long as her last shot is 12 hours or more before surgery, she can go into the operating room without any blood thinners in her system.

After surgery, Jo starts back on Coumadin, but it takes 5 days to build up. She again gives herself Lovonox shots for 5 days to “bridge” the gap until the Coumadin is built up. This “bridge” is extremely important. A total hip replace is a very significant surgery with a large incision and a lot of potential for bleeding. Jo’s surgeon, Dr. Kelly, had an extremely delicate job to do in managing Jo’s recovery because thin blood was necessary to prevent clots, but keeping Jo’s blood too thin could also cause problems with the healing process of Jo’s wound.

After checking as many records as possible, it appears that Jo was given 2 Lovonox shots in the hospital after that hip replacement. It is very possible that she went through a short period of time where her blood was not as thin as her hematologist would have liked it and this could be when the clot was formed. We’ll never know for sure, but the NIH doctors agree that there is enough data to suggest a reason for the clot. The other scenario would be to find that the clot happened even though Jo was fully “juiced-up” on blood thinners – Dr. Alvarez calls that “therapeutic”. I have no idea what that means. If that were the case, we would already be making plans for a bone marrow transplant.

Yes, Jo does have the PNH problem, but relative to other PNH patients she has it easy. She does not require any transfusions, she only had a handful of hemolytic episodes in the past year (lot’s of red cells bursting) and most of her hemolytic episodes have been fairly light. Therefore, after 2 weeks of discussions we have chosen to stay the course.

The other thing brought up after we talked about the blood clot was the possible use of Soliris – the “miracle drug” that was approved by the FDA last March. PNH patients that take Soliris do appear to experience blood clots less often than patients that do not take it. On the other hand, Soliris also has its issues. First, it is a probable commitment for life and there is no data yet on the long-term effects of taking it. Dr. Alvarez knows of one patient taking it here in the Denver area and after a couple of years now, that patient is beginning to have mixed results. Secondly, the drug costs $480,000 per year. Thirdly, most of the people (maybe all) have PNH problems more severe than Jo’s.

The question becomes this, “If Jo’s blood clot can be blamed on her level of blood thinners after her surgery, is her PNH condition bad enough to warrant the use of Soliris?” I will tell you that the opinions are not unanimous.

Considering Jo has felt tremendous the past 12 months, it seems odd to even have had this discussion over the last 2 weeks. It has made us realize how narrow the tight rope really is. We are very fortunate to have the medical advisors that we do have. Primarily because of how good Jo is doing, we have decided to push forward without any major changes. There are some minor changes and I’ll explain those later.

On a side note, have you ever seen the 1976 Movie, “The Boy in the Plastic Bubble”? John Travolta played the “Bubble Boy”. (I know that most of you are probably more familiar with the Bubble Boy episode from Seinfeld.) The bubble boy was the son of hematologist at the NIH and he developed Aplastic Anemia. The progress made with this disease over the past 30 years has been absolutely incredible. The really neat part is that we found out that Dr. Alvarez was a resident at the NIH 30 years ago and he was actually assigned now and then to take care of the Bubble Boy! Small world, eh?