Male1: It has been done for about 50 years now, is it not? Male2: The prophylaxis with penicillin--it is close to that. I mean it has been for a very long time, there was a very big study in the very early 80 that essentially established it as the way to go for this particular disorder, and one of the things that it is important to note is that kids with sickle-cell disease used to die very commonly prior to this, while instead death in the first 20 years in life in patients with sickle-cell disease is fortunately a rare event. It still exists at a level that are higher than the general population, but it is a rare event. Male1: So as long as they are getting penicillin and giving the kid a vaccine againts the pneumococcus, what other things are getting treated? Male2: Well, there are many things that one needs to do, for instance, one of the most devastating complications of sickle-cell disease is something called a stroke. The sickle-cell disease affects the blood vessels among other things and young kids with sickle-cell disease may have stroke. There is a significant incidence of this problem. A stroke is essentially a plugging and a cohesion. Male1: And what can we do to prevent that complication? Male2: Well, interestingly enough, in the last ten years, a lot of study has gone into this and there is very good evidence and very good study that say that measuring the blood flow in some critical blood vessels in the brain through something called a trans-cranial doppler which sounds terrible, but it is actually a very simple and not invasive procedure. It is essentially an ultrasound of the blood vessels and we ultrasound for all kinds of things. That actually gives us a very good idea whether or not a person is at risk for the stroke or not with patients with sickle cell and those patients who are at risk will actually decrease their risks substantially if they are put on a chronic transfusion treatment. So tranfusions in those patients actually are the accepted way to essentially reduce or negate the risk for stroke. Male1: So if they get a lot of transfusions, we still run a risk of getting-- Male2: That is the down side of what we have in life. In medicine, there is no free lunch served. Male1: And we have a way of getting that out of the body. Male2: And these patients, once they go in chronic transfusion will require what we call the iron chelation therapy, which is essentially therapy to reduce their iron in the body and with the new chelators, the patients with sickle cell disease can benefit from that and we are actually having very good results in the sickle cell disease as well as in Thalassemia with the new oral chelators. Male2: And down the road, bone marrow transplant is a--? Male2: Bone marrow transplant is already a possibility in sickle-cell disease. For a variety of reasons and because the disease is so heterogenous, meaning it has so many different manifestations and it is so benign in some patients that it is so devastating in others has not been used across the board for these patients, but we know that patients with sickle-cell disease can be cured of their disease with bone marrow transplant and there are at this point, probably several hundred patients who have been transplanted here in the year. A little anecdote, the first patients with sickle-cell disease was actually transplanted because the patient had recurrent leukemia and the patient happened to be treated and cured with both sickle-cell and leukemia and I believe she is still alive today, many years from. Male1: So there is great hope down the road? Male2: It is great hope in this disease, also there are for instance, other interventions to the change in the face of sickle-cell including the use of hydroxyurea. Hydroxyurea is a medication that was used actually as cancer treatment for a long time, but has a very good track record of being used in the chronic and the long term kind of situation and without significant side effects and there was a very pivotal study about--it is getting close to 15 years where hydroxyurea was used to see whether or not it could prevent some of the complications of sickle-cell and it does, and a number of patients nowadays both adults and the adolescent patients are on hydroxyurea and that has made their life incredibly better. Male1: Thank you.
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