Sickle Cell Disease: Diagnoses, Screening, and Treatment - Episode 6

Hematopoietic Stem Cell Transplant in SCD

January 20, 2020

Peter Salgo, MD: I tell you what, I’m going to cure everybody. That’s it, everybody is going to get hematopoietic stem cell transplantation. Boom, we’re done! Good idea? Bad idea? Where do we go with this?

Biree Andemariam, MD: I think in a select population, particularly in pediatrics where most of the literature is right now, there are probably, what is it, maybe 2000 or 3000, maybe more babies that have been….

Jane Hankins, MD, MS: Two thousand.

Biree Andemariam, MD: Two thousand who have been transplanted worldwide at this point.

Jane Hankins, MD, MS: Oh no, 1000 people have been transplanted with sickle cell disease.

Sophie Lanzkron, MD, MHS: So, it’s a 95% survival rate.

Biree Andemariam, MD: Yes, it’s success.

Sophie Lanzkron, MD, MHS: And 90% disease-free survival.

Jane Hankins, MD, MS: If you have a matching sibling, right?

Biree Andemariam, MD: Yes.

Sophie Lanzkron, MD, MHS: Under the age of 13.

Peter Salgo, MD: Let me hear these numbers, 90%, 95% survival?

Biree Andemariam, MD: Yes.

Peter Salgo, MD: Of those who survive, 90% disease-free. Is it fair to use a powerful word, are these kids cured?

Biree Andemariam, MD: Yes, but let me reverse that. I’m a mother. What I heard was there’s a 1 in 20 chance that my child might die. That’s what I heard. But there’s also a strong possibility that my child may live into their 40s.

Peter Salgo, MD: Without the transplant.

Biree Andemariam, MD: Without the transplant.

Jane Hankins, MD, MS: Without the transplant.

Biree Andemariam, MD: And again, I’m not a pediatric hematologist, but I am a parent and that’s what I hear, and I think that’s the challenge when we’re dealing with sickle cell disease, a so-called nonmalignant disease where the lifespan of the child in front of you is not easy to determine. You just can’t see into the future. It’s not like having a baby with leukemia. I don’t know if you guys disagree with that.

Elliot Vichinsky, MD: I don’t agree with that, but I do agree with, I think parents need to make their own decisions.

Jane Hankins, MD, MS: I agree.

Elliot Vichinsky, MD: But I do think it’s a progressive bad disease, and if you had a full HLA [human leukocyte antigen] match, which most people with sickle cell don’t, those results come from when you have an HLA matched sibling, and you have a young child, and then you have low risk factors. And knowing how bad adults, the quality of life, I think if you identify a patient with the disease, having the family have the option to do that. The physician doesn’t make those decisions but offering the family in the menu of things that could be done would be an appropriate thing to do.

Peter Salgo, MD: Can you do a transplant without an HLA identical sibling?

Jane Hankins, MD, MS: Yes.

Biree Andemariam, MD: You can.

Peter Salgo, MD: But I’m assuming the risk is higher.

Jane Hankins, MD, MS: And the results are not as good.

Peter Salgo, MD: And the failures are higher.

Jane Hankins, MD, MS: Yes.

Peter Salgo, MD: So, when would you consider that as an appropriate therapy?

Jane Hankins, MD, MS: For those, because the risk of rejection, of graph versus host disease and mortality, because they’re all higher, I have a very heart-to-heart conversation with my parents. And I say, “Go for it if you’re willing to take the risk because the risk of not having it is high enough.” For example, I have had a stroke, I’m about to have a second stroke, and a third stroke, I’ve had those conversations. I had a patient who had 3 strokes and he had brain revascularization, and he was on hydroxyurea, and transfusions, and aspirin. And he kept having micro-strokes. And I said, “You don’t have a donor.” You have to think here he may have a fourth and that may be it for this child, so they decided to go through with the haploidentical transplant.

Peter Salgo, MD: Does that clinical scenario where the child’s had multiple strokes and multiple other comorbidities put them at higher risk for the transplant?

Jane Hankins, MD, MS: Probably, but the disease will probably be just as high.

Peter Salgo, MD: Where I was going was would you wait for all of those complications or transplant earlier?

Jane Hankins, MD, MS: The way I think about sickle cell disease when I have discussions with my parents, I say this is like a college fund. Your child is born. Today you’re putting money into this account, and putting money in this account, for me, is disease-modifying therapy. So I give all the information when they’re born—hydroxyurea early in life, if you need transfusions, we’ll transfuse and start thinking about curative treatments.

Peter Salgo, MD: Now, to put this in the popular context, especially in somebody whose life expectancy is probably in the mid-40s for whatever reason, it’s almost a Sophie’s choice. How do you make this choice for your child? I personally have 3 children. They get a hang nail and I fall apart. I don’t understand how parents can....

Biree Andemariam, MD: We haven’t even talked about fertility.

Jane Hankins, MD, MS: That’s right.

Peter Salgo, MD: Yes.

Elliot Vichinsky, MD: I think you don’t make the choice, number 1. You offer families options.

Jane Hankins, MD, MS: Yes.

Peter Salgo, MD: How does a family make that decision?

Elliot Vichinsky, MD: I think it’s important that while the HLA-matched sibling transplant is, to me, an accepted standard for parents to get, there are a lot of other newer developing options for young adults and others to have transplant-like therapy, but they should only be done in really validated studies. And that’s where we need to enroll patients to answer those questions, and there are trials open. So, if families are interested or knowledgeable, they should not be done in local areas by themselves. They should be part of a larger study to be able to answer the questions.

Transcript edited for clarity.


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