Emerging Therapies in the Management of Sickle Cell Disease - Episode 8

Patients Considered for HSCT

Biree Andemariam, MD: We’ve talked a bit about long-term or existent therapies that have been around for at least a few years, if not a few decades. Julie, segue now over to curative approaches. Which patients would you consider for curative hematopoietic stem cell transplantation [HSCT] and why?

Julie Kanter, MD: Thank you. I had the honor recently—and hopefully we’ll all get to share in the American Society of Hematology’s HSCT guidelines. We’ve put the paper together, and I’ve had the honor of reviewing all of this recently. I think the data are most strong and incredibly clear-cut for individuals with hemoglobin SS or S-beta-0 disease who have a matched sibling—especially those at risk for stroke, whether that’s primary or secondary—to undergo stem cell transplant. The outcomes are amazing: 95% event-free survival in this group of patients, especially those under 13 years old, and that’s with myeloablative transplants.

That’s the first group. I recommend anybody who has a patient or a child with a high stroke risk who may have a matched sibling to undergo typing. As we start to look at slightly older patients, we start to have to ask a few more questions about what disease things we can and can’t reverse. There are complications of sickle cell disease, like bad avascular necrosis, that we can’t reverse.

It’s important when we approach a curative therapy like stem cell transplant—our only known curative therapy at this time—to make sure we’re asking the right questions. We need to see what the patients hope to gain and always make sure they see a sickle cell specialist before they undergo a transplant conversation, so that they can have a shared decision-making conversation about pros and cons. There are amazing data coming out of nonmyeloablative transplants for siblings in the adult population, as well as for haploidentical or half-match transplants. I think we have some really exciting opportunities on the horizon.

Elliott Vichinsky, MD: I agree. I think transplant’s progression is wonderful, but It’s important as a community that we have good long-term data on its effects in terms of diabetes, secondary malignancy, and lung injury recurrence. It’s exciting, but as a group, we need to collect that data.

Julie Kanter, MD: Elliott, that’s so important because that was the hardest part about writing this paper. We don’t have so many data that we need that are gathered in a uniform way. It’s why we desperately need a clinical longitudinal registry that can look at both transplant data as well as data about these new therapies we’re going to talk about. We have to be able to compare things. One of the NASEM [National Academies of Sciences, Engineering, and Medicine] recommendations was an ongoing clinical longitudinal registry, which I think is what we are missing to answer so many of these questions.

Transcript Edited for Clarity