Dr Lametra Scott on the Standard of Care and Unmet Needs in SCD

Transcript

What is today's standard of care for SCD? And what standards of care still warrant intervention?

Today's standard of care is hydroxyurea or a blood transfusion—hydroxyurea for those who have sickle cell anemia, meaning that they have the genome type of sickle cell type SS, or sickle beta zero. And for those particular situations, those warrant no hemoglobin A production at all. Hemoglobin A is normal, and it does not sickle. In sickle cell SS or sickle beta zero, the body does not make hemoglobin A, it only makes hemoglobin S. And in that regard, hydroxyurea comes on board because it actually causes the body to produce increased amounts of hemoglobin F. Hemoglobin F stands for fetal hemoglobin, and that's basically what's made during the infancy stage. And hydroxyurea turns that hemoglobin F signal back on and hemoglobin F is important because it does not sickle. So for those who have other genotypes other than SS or sickle beta zero, hydroxyurea, it is negligible whether or not it can provide the great benefits of reproducing that hemoglobin F that does not sickle. So for those patients, we have blood transfusions and blood transfusions are not for every person, but it's for those who have an increased stroke risk. You want to make sure that you prevent that. It’s for primary and secondary prevention of stroke, or ot's for someone who has multisystem organ damage, or organ failure is beginning to come aboard. And that's where a blood transfusion would be warranted, or it's for someone who has an emergent need, where they are having a dramatic drop in their hemoglobin levels that may be brought about because of an infection or because they are maybe having surgery. For whatever reason, they may need a blood transfusion.

Blood transfusion, while it is available, is not applicable to every sickle cell patient because taking on a blood transfusion, over time, it causes situations in secondary complications in the body as well; [it] would need to be warranted. And it's not something that you would want to just use as a go-to. So where we are right now in the unmet needs and what we have? As you've heard, there are limitations with the current standards of therapy. We still have needs [regarding] what do we do for these patients that don't qualify for the standard of care. So that's where we are right now. Another unmet need is, in addition to not having access to current treatment options, patients don't have access to actual providers, and that's because there's a lack of specialists to treat sickle cell disease. So the issue is compounded for sickle cell patients in terms of what the unmet needs are. We have lack of access to care, lack of access to providers, lack of utilization. So even though the treatments are there, they're not being utilized as they should be, so that we can decrease the sequalae and improve patient care with what we have available.