BSH 2023 | Optimizing care and improving quality of life in pediatric patients with sickle cell disease

So, we had a really good discussion about that in the Meet the Expert session. So, it was mainly to do with really trying to improve the care of pediatric sickle cell disease. We actually discussed the use, and how we can improve the use of hydroxyurea in this context. There are significant lessons to be learned from more recent research, a very interesting study which was conducted in Africa which we can draw a great deal of information from. It was the REACH study which was published in the New England Journal of Medicine a couple of years ago, and it clearly showed that the superiority of a maximum tolerated dose regimen compared to a fixed dose regimen. It was clearly in the African context it worked very well and that kind of led us to really push the MTD, the maximum tolerated dose regimen and increase the uptake of that MTD regimen type treatment in sickle cell disease, in the UK. I know that a lot of other centers in the U.S do that already, but I think there is a little bit of reluctance to proceed with that in Europe and I think this study has really helped change the minds. The other thing we discussed about how soon we can start the hydroxyurea treatment in children, and it was discussed whether nine months, you know, whether people are doing it at nine months or whether we are doing it a little bit older, at maybe age one year or 18 months and so on. So, it was quite a sort of rich discussion we had in that session about how to improve the access of hydroxyurea to all children with sickle cell anemia and also how we can reduce the kind of skepticism that it might be prevalent both amongst doctors, nurses as well as among parents. So that was quite an interesting talk. The other thing which I thought was very rich, the discussion that came out of that session was how we really should try and improve or raise the bar of treatment that we have at the moment, its mortality that we are kind of looking at and how we kind of tend to be really happy that children are no longer dying in childhood in the Western countries. But I think we should concentrate not just on mortality but quality of life and preservation of organ function. So, I think in that sense, hydroxyurea is a very valuable tool to do. So that’s kind of mainly the thing and I have had people come back to me to say that message to start hydroxyurea really early and aim for maximum tolerated dose has hit home really well and that will have changed their practice going back to their clinics.