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Stem Cell Transplant: What Is It and When Is It Appropriate?

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Published on July 23, 2014

What is stem cell transplant? When should this be considered as a treatment option? MPN experts, Dr. Bart Scott from Seattle Cancer Care Alliance and Dr. David Snyder from City of Hope, share their knowledge and insight about stem cell transplant. Dr. Scott describes the origin of this type of treatment, while Dr. Snyder discusses how candidates for stem cell transplant are determined and when this treatment should be considered.

This event was produced in association with City of Hope and sponsored by Patient Empowerment Network through educational grants from Incyte Corporation and Geron Corporation.

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Transcript | Stem Cell Transplant: What Is It and When Is It Appropriate?

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

Andrew Schorr:

So what is a stem cell transplant? Michael has had one. We’re going to talk more about that. Where does that come into play, and what is it?

Dr. Scott:

Okay. Well, the what is it question I think is the easier one, so I’ll go with that one first. So, before I had anything to do with medicine, back in the early ‘60s, there was experimentation with giving high doses of chemotherapy. The idea was that if a little bit of chemotherapy kills cancer, then higher doses of chemotherapy will kill more cancer.

So that’s not that revolutionary of an idea. That makes sense to all of us. But they found that a lot of patients were dying from bone marrow failure. Because if you look at different types of chemotherapy that we give for cancer, by far for the majority of those drugs, the dose limiting toxicity is stem cell poisoning. So it can poison your stem cells.

The stem cells die, and these patients developed what we call aplasia. Basically, they stopped making blood. And so initially, stem cell transplants were developed as a way to rescue patients from the side effects of high doses of chemotherapy. But there was some, later on some data that came out.

That basically showed that stem cells’ transplants also work by giving you a new immune system. And this new immune system can recognize the cancer cells as being foreign and attack them. So just like if you get an infection, you get a bacterial infection, your immune system reacts against it.

If we give you a new immune system, this new immune system can attack that cancer. We use a variety of different terms for it: GVL, GVT. But it stands for graft-versus-leukemia or graft-versus-tumor effect. So we now know that transplants work in two ways. It works by the conditioning regimen that we give, before the infusion of the stem cells.

And it also works through this allograft effect. And so later on, now, we’re starting to reduce the intensity of the conditioning regimen before the infusion of the stem cells, and we are relying more heavily upon this graft-versus-leukemia effect. So that’s essentially what stem cell transplant is and how it works.

 

Andrew Schorr:

Okay. Let me ask Dr. Snyder. How do you determine who needs one?

Dr. Snyder:

That’s, that’s a…

Dr. Scott:

That’s the harder question.

Andrew Schorr:

I give him the hard one.

Dr. Snyder:

Right, right. No, it’s a very complex equation to consider. And like with any medical decision I’m making, it’s a balance of the risks and benefits of one alternative, versus the risks and benefits of another. So we’re going to talk about, you know, ruxolutinib (Jakafi) and other new drugs that are coming along and research that’s being done.

But still currently, an allogeneic stem cell transplant is the only known way to cure these diseases. So if that’s something that is of importance to the patient, to the family, something as a goal, the only way to get there is through a stem cell transplant. But, of course, it’s not so easy, you know, to get there.

There are definitely serious risks associated with it, including dying earlier than you might if you didn’t go through a transplant. And as we talked about, even if you survive, there may be issues that compromise quality of life—not necessarily quantity, but quality of life, in particular something called graft-versus-host disease.

So it is a complex decision. But for me, when a patient with myelofibrosis, either primary or secondary has reached a stage, the intermediate two or high risk, let’s say they’re requiring frequent red cell transfusions, the quality of life is compromised. That’s the time to consider is this person a candidate for a transplant?

And there are a lot of things you have to look at. One is the age of the patient. Fortunately, because of things like reduced intensity conditioning, now we can raise the age. Instead of 50, 55, we can talk about someone 70, 75 as a potential candidate. What other comorbidities does the patient have?

Other diseases, diabetes, hypertension, etc. Is there a donor available? Possibly a sibling donor, which would be the first place we’d look. But if not, to look in what’s called the National Marrow Donor Program registry to find an unrelated donor. And nowadays, possibly expanding to cord blood and even half-matched donors. So that’s a factor as well.

So my approach is to raise this option with a patient, when we see the disease status starting to move on to these more advanced stages—but not necessarily to move right away to the transplant. That would be the time to explore the option, look into available donors. But I don’t like to interfere with good quality life that my patients are enjoying.

So if they’re able to work daily and get home and still have some energy left at the end of the day or over the weekend, I don’t want to interfere with that. But if they tell me, you know what? I can’t do my work.

I can’t get through my work week anymore, or I get through my day, and I just crash when I get home. I’ve got to do something different. So that’s the time to say, all right. We need to move ahead to transplant.

Andrew Schorr:

Okay, anything you want to add, Dr. Scott?

Dr. Scott:

I agree with everything he said. It’s a balance between patient risk factors and disease risk factors, and it’s a complicated decision, and you would really need to see a transplant specialist to help you with that decision.

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

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