Tuesday, September 16, 2008

My visit to the NCI, Part 1

Marilyn and I like road trips. We’ve taken them for as long as we’ve had something with four wheels and an engine.

In the old days we went car camping out of ou
r Toyota Celica, pitching a backpacking tent in Death Valley, which threatened to blow away during a dust storm -- with us in it. Then we upgraded to a Volkswagen Westfalia van with a pop-up roof, in which we went over the Rockies in first gear. Our cat, Tut, used to nap on a picnic basket wedged between the two front seats. This was followed by the RV era, which involved towing a house (aka fifth-wheel trailer) behind us. Now we’re back to square one -- just a car -- though we prefer motels to tents these days.

And now that I have chronic lymphocytic leukemia, we’ve combined ro
ad tripping with doctor visiting. In 2006, we set out for Columbus, OH, home of Dr. John Byrd. And in July of this year we took off for Bethesda, MD, home of the National Cancer Institute and National Institutes of Health.

We were going because I was looking into a clinical trial that offers free reduced-intensity- conditioning stem cell transplants using matched unrelated donors -- the popular mini-MUD. I need an unrelated donor because I have n
o sibling donors; my two brothers and one sister are all half-siblings. Since my health insurance won’t cover transplants, participating would be like winning a million-dollar lottery of sorts. Of course, I’d rather win a million dollars and not have CLL, but that’s another story.

CLL Topics published an article about the trial in January, one mont
h after I completed my R-C(V)P therapy for CLL and autoimmune hemolytic anemia (AIHA). The year 2007 had not been kind to me: AIHA was diagnosed in March, treated, and followed by relapses in July and October, each worse than the one before. Not only was my quality of life going down the tubes, it appeared the AIHA might be uncontrollable. The idea of a transplant, which had once been fuzzy and distant, was now taking on some immediacy. It was prudent to look into it seriously, especially as I had no idea in January how long my remission might last.

One in seven million

So I called the transplant coordinator and explained my situation. He said I was, on the face of it, worthy of consideration.

The first step would be to see whether there was a 10/10 HLA match, which is required for participation. (A less-than-optimal match equals less-than-optimal results, both for me and the trial.) A blood kit with two tubes was sent from Bethesda. I had the blood drawn at a local lab and then Fed-Exed the package back to the NCI, along with copies of my medical records.

It took several months to get any answers, partly because the trial was stopped at one point to allow for a routi
ne review of how the protocol was working. It turned out that the six people who had undergone transplants were doing well, and so accrual of patients resumed. A total of 20 people will eventually be enrolled.

The NCI first looked at my blood on low resolution for 6/6 matches, and when a number of those were confirmed by searching worldwide donor databases, moved onto the next step: testing at low resolution for 10/10 matches. Twelve were found, and this was followed by the final search at high resolution, after which the tr
ansplant coordinator called with the news: Of the twelve 10/10 low-res matches, only one donor, out of seven million in the databases, appeared to be a 10/10 match at high res.

But one, as he pointed out, is all it takes.

He added that the ne
xt step in testing, should I be provisionally accepted into the trial, would be to contact the donor and make sure he or she had no infectious diseases and was physically able to undergo the donation procedure.

The testing and donor searching that had already been done -- which would have cost me $15,000 or more had I been paying for it myself -- thus answered a very important question: Are there donors for me and is an adult stem cell transplant therefore going to be an option now or in the future?

The answer is -- and it’s fortunate that I am not a Type A personality -- “maybe.” It is worth noting here that the 5-million-strong U.S. National Marrow Donor Program database returned 22 low-res 6/6 matches when I used their website's HL
A matching gizmo last year. In retrospect, those results are pretty much meaningless. When the rubber meets the road in the form of further testing, 22 6/6 matches can mean bupkus, or close to bupkus. (For those unfamiliar with road trips, Bupkus is just east of WaKeeney, in central Kansas.)

Having completed the HLA preliminaries, the transplant coordinator wanted to move ahead to the next step, an “informational meeting” with one of the doctors involved in the trial. I could have done this by phone, but I hate talking on the phone. You can’t see who you’re dealing with, you don’t have the presence of mind that you do when you’re there with all five senses, and you can’t grok the facility,
get an impression of what actually taking such a big step would be like. So off we went.

Did I mention that this was also a good excuse for a road trip?

Meanwhile, back at CBC ranch

In the months following my three rounds of R-C(V)P, my CBC continued to look better and better. Not only had the hemolysis stopped, but my red counts were making a slow climb toward normal. My lymphocyte count was c
ontinuing to drift downward. My lymph nodes, which were knocked back to the point of being less swollen than they were at diagnosis in 2003, were stable. By the time we went to Bethesda, my red counts had crossed the line into the low-normal range and my Coombs test, though still positive, was now only “microscopically” so. The nodes had come back a little, but not much. My spleen and liver were behaving themselves. The lymphocyte count had dropped to its lowest level since I had the chemo eight months before.

In other words, I was doing better than I could have hoped when I finished the R-C(V)P in December and approached the NCI in January.
And so the transplant immediacy factor was beginning to diminish. When your back is against the wall, you do what you have to do, regardless of the risk. When the pressure is off, you look at things from a different perspective, especially when you are considering taking a step that has a 15% to 20% chance of killing you.

But the prospect of having just one donor added a little drama to my decision as we traveled east. Do I seize the moment before there is no donor at all? Or would it be reasonable to expect that more donors might come along in the future

And are those even the right issues to be focusing on?

For as a learned friend once told me when I was young and we were all sitting around a campfire drinking a lot of beer, “The thing in life is not to know all the answers but rather to ask the right questions."

In Part 2, I will discuss the questions I asked, the answers I found, and the general consensus to date when experts grapple with the question, "Who should have a transplant when?"

We stopped to see the giant Van Gogh sunflower canvas in Greatland, KS. Apparently Van Gogh was taller than we thought and traveled further afield than is commonly known. The middle photo is of our cat Pyewacket in the driver's seat on our (doctor-visitless) road trip to Nova Scotia in 2004. Both of our cats have enjoyed traveling; we adopted Pye at an RV park. The top photo shows Tut enjoying a claw-scratch at a campground, circa 1987. That's a 1971 VW.


Anonymous said...

Hi David,

Before I would undergo a transplant, I would want to be put into contact with several people who had had the same procedure and who were in good health for at least a couple of years after. And having to take drugs that made me feel lousy in order to stay alive after the transplant doesn't count. It is a fact that a certain small percentage of people with CLL have unexpected long-term remissions even years into their CLL fight (I'm one of them). You have to balance that ever-present possibility against the rather great chance that a transplant is going to leave you worse off (perhaps as much worse off as it's possible to be) than you would have been without it. I could be wrong, but my gestalt impression is that transplant doctors tend to paint a more hopeful picture than can be justified by actual outcomes.

What do you think?


David Arenson said...

I know of many transplant cases from CLL Forum, ACOR, and patient blogs. In the past year two friends have undergone transplant: one died unexpectedly, the other is doing well beyond all expectations. So for me the lesson is that the results can be arbitrary.

To undergo a transplant is to play with fire. The reward is, perhaps, a cure; the flip side is getting burned in any number of ways. Death is not always one of them. A patient I know of has developed chronic GVHD and some two years after the transplant scleroderma has set in. Her skin has become like hard plastic. It is not pleasant. Dr. Terry Hamblin is one of those who is less gung-ho about transplants; he knows of two patients who killed themselves due to difficult GVHD issues.

At the same time, I could go on and on about successful cases, or cases where problems remain but those problems pale in comparison to what the alternative might have been (death).

I am also aware of cases like yours, where the disease does not take the expected course. Such cases are rare, but unexpected outcomes are to be expected in medicine. And so I do think there is wisdom in exhausting all other avenues -- even ones that have not been proven in peer-reviewed multi-center trials, such as exercise, or such as TCM (Traditional Chinese Medicine). You never know when you might get lucky.

But it is important to remember that most cases play by the book. So it is essential to plan ahead, be ready for whatever step you need to take. Thus one reason for our trip to Bethesda (which, to bring up the "unexpected" point again, was taken against the backdrop of an unexpectedly better remission from chemo).

As to transplant docs painting a more hopeful picture than can be justified: I am sure some do. They are dealing with sick patients who may be embarking on their last chance at survival. So I think there is a tendency to accentuate the positive. This is also the doctors' line of work, and they probably have seem improvement over time, so they may have a general sense of progress even if an individual's response may be different. You also have the statistics -- chances of success are better than chances of failure. (There's about one-fifth chance of death, one-fifth relapse, three-fifths cure or long-term remission.) So on the face of it there is reason to be more optimistic than not.

Still, none of us knows what the fickle finger of Fate has in store. The doctors have not lived the procedure; they have only performed it. What the patient experiences is entirely up to chance. And it's a big chance to take.

Laisha said...
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