Is a BMT inevitable?

[KimO]

My daughter had her hematology appt today and once again the doc expressed his opinion that a BMT would be required. This depite the fact that her blood counts today were Plt 196, ANC 1.85 and Hgb up this week from 9.7 to 10.2. She is hoping to graduate from UW after the winter quarter and he apparently talked about most students heading out to find jobs after graduating but not her. I don't disagree that a consultation with the Fred Hutchinson center might be a good idea, but am I mistaken in thinking that even considering a BMT when her counts are recovering so well on cyclosporine alone is crazy?

I'd also appreciate opinions on whether it will ever be possible for her to wean from the cyclo (assuming VERY slow and starting after she's been at CR for awhile) since she relapsed after being off for a year.

[Neil Cuadra]

You've got the right questions, Kim, but it's hard for us patients and caregivers, each with our own experiences, to second-guess Shauna's situation. However, we can give you opinions. A transplant is a reasonable treatment choice because she's young (which means that she's a statistically good transplant risk) and because it's a chance for a cure (nobody wants AA hanging over them with their whole life ahead of them). But I wouldn't think it's "inevitable" since wait-and-watch would also seem to be a reasonable choice, especially since Shauna achieved remission for over 2 years before, and is doing OK right now. Repeated ATG treatments, including those given many years apart, can be all that's needed for some patients. It's a big decision, whether to stick with what you've got or accept the risks of a transplant for the chance to get a permanent cure.

When my wife and I faced that decision, second opinions were invaluable. I suggest that you get a second opinion if you can so you'll have as much information as possible.

The doctors may not be able to predict if Shauna can get off cyclosporin completely, but if she was able to get off of it last time, perhaps she can do it again, or at least get down to a very low dose.

[Mary4Mike]

A consultation with a transplant center is a good place to go for another opinion. When my husband was first diagnosed, and 4 or 5 years into the disease, we consulted with a transplant center. THEY told us that he was not yet a candidate for transplant because of decent counts no blasts, etc. Ultimately that changed (10 years later) and he did have a transplant. It does seem though that recently, transplant is given as an option soon after diagnosis. Having said that, there was no pressure to transplant. Your options are laid out for you and it is ALL your decision. It is a wonderful education process to talk to a transplant doctor even if that never becomes a reality for you.
All the best to your daughter and you.

[Greg H]

Hey Kim!

I second what the other have said and would add that different docs can have very, very different assessments of the best treatment option. I really think you have to listen to all of them, understand why they are recommending what they are recommending, and then (to paraphrase a former POTUS) you are the "decider."

I have huge respect for my transplant center doc at Wake Forest, and a huge amount of respect for the NIH doc running the trial that I'm about the enter.

The former -- backed up by her whole team, including the docs specializing in MDS drug therapy -- felt I should have a transplant now, despite minimal quality of life impact of my anemia, minimal blasts, etc.

The NIH doc said, given my chromosomes and low risk profile, if I chose not to participate in his clinical trial, he would recommend that I watch and wait.

My local hematologist wisely observed: "When you're a hammer, everything looks like a nail."

For me, I can't just weigh the opinions and credentials of those two fantastic docs, or flip a coin. I have to understand why each of them is recommending what they are recommending and then decide where to place my bets.

Good luck with your decision.

Take care!

Greg

[Lisa V]

I agree with the others that consulting with a transplant doctor before making any decisions would be the wisest thing. They really do have a better perspective on the issues involved than the average hem/onc.

I remember our doctor walking in one time a few months after Ken's second ATG and stating that in his opinion we'd "waited long enough", and that he was going to send his files off to the City of Hope team for evaluation. This came as something of a shock to us, as he had been showing gradual improvement, and we've always wanted to avoid going to transplant if at all possible. He even said he "couldn't imagine" them not agreeing with him. Well, at the next appointment he told us that CoH had reviewed his case and felt that a BMT was not appropriate at this time. End of story, no further comment. Yay!!!!

Ken's counts have continued to improve, so I feel it was the right call. On the other hand, there is the cyclosporine dependency issue, which is a concern. I don't think any of us here can predict whether or not Shauna will be able to successfully discontinue the cyclo long-term or not. The trend now seems to be towards longer, slower tapers, along with the growing realization that some people will have to stay on it indefinitely. This is all fairly new thinking, though, and medical opinions continue to change as they learn more.

[Hopeful]

I am also in agreement that now (while *not* in crisis mode) is the time to consult with a transplant doctor.

Shauna's case is unique because she is young at 22, but not so young that a BMT is the obvious first line of treatment. Everyone knows that IST is not a cure, but is does buy time. Time for more research to be developed that may find the ideal BMT regimen or maybe even the miracle drug-cure. Advancements are coming at such a rapid pace right now.

The tradeoff though is that while you are buying time with IST, Shauna is also getting older. So, the risks of a BMT increase. She also may want to have a baby at some point, which again increases the risk of relapse.

But why the urgency to do it right now? Why not ride the IST tide for a few years and enjoy life as much as you can... It is a tough call, which is why the consultation is so important.

[Lisa V]

Originally Posted by Hopeful

Everyone knows that IST is not a cure, but is does buy time.

Generally speaking, this is true, but the figures I've seen for IST cite a relapse rate of roughly 35% at 14 years, for both children and adults. If I interpret that correctly, that means that 65% of responders will NOT relapse, at least not within the time period measured. I don't know of any statistics for longer periods of time, but if there were, they would probably be outdated since protocols and methods are constantly being improved.

Also, it is not unheard of for a patient to relapse following a BMT. I've heard of at least a couple of cases of that happening. The bottom line is that nothing is absolute. We're always dealing with odds and statistics and trying to second guess where we fit into the picture. The best people to help with that are those on the frontlines, but it's still going to be an educated guess at best.

[KimO]

Thank you so much for all the well thought out replies. It's definitely got me looking at the issue in a different way. Shauna has an appointment next month with her doc here at home and we'll talk to her about getting a consult with the Hutch. I know the doc in Seattle called them when Shauna first relapsed and they were the ones that suggested Cyclosporine alone and then a repeat ATG if that did not start showing results in three or four weeks. It surprised me a little then since I assumed that they would be biased toward transplant. They also know the quality of the matches that were identified three years ago -- all we know is that they found a match. As you have all said, it's just better to talk to them directly to understand their recommendations. Thanks again for providing a resource. As we all know, there aren't very many people available that you can bounce things off of that truly understand.

[Hopeful]

Originally Posted by Lisa V

Generally speaking, this is true, but the figures I've seen for IST cite a relapse rate of roughly 35% at 14 years, for both children and adults. If I interpret that correctly, that means that 65% of responders will NOT relapse, at least not within the time period measured. I don't know of any statistics for longer periods of time, but if there were, they would probably be outdated since protocols and methods are constantly being improved.

Hi Lisa,

My comment was more with regards to 10 year survival than to relapse. The papers that I have read put 10 year survival for IST at 73-75% for patients younger than 30, 66% for patients 31-40, and 47% for patients older than 40 years. I don't know if these numbers include responders and non-responders. I've asked my doctor what happens if you make it the 10 years, but he only emphasized that in my age group only 47% do

[Lisa V]

Therein lies the problem with statistics, Hopeful. It's hard not to read more into them than is there, or make certain assumptions that may not be warranted. In articles that cite the 35% relapse rate, I have seen no mention of where non-survivers fit into the picture or how that skews the percentage. I was assuming that death would be preceded by a relapse, but of course there could be other scenarios too.

I guess the point I was trying to make is: if the majority of responders to IST will not have a relapse of their AA, that seems pretty close to a "cure" for those people, regardless of their age. Of course not all of them will return to full normal counts, so maybe that's the distinction. However, when you throw in GVHD and all of the other baggage that goes with a transplant, it's hard to see it as a clear-cut case of one being a total cure that you can walk away from with no issues, while the other isn't.