General Updates | Examining the early-life clinical and hematological profiles of patients with sickle cell disease

So, sickle cell disease, as we know, is a genetic disorder, so babies are born with the disease. And if you look at the profiles, the hematological profiles of babies with sickle cell disease and those without, one of the key things that we have been interested to look at is the fetal hemoglobin levels profile and how these are different between the children with sickle cell disease and those without. And it’s really key because we always say that this is the crucial point which actually defines the disease itself. Because when they are born at birth and when the levels of hemoglobin are high, you don’t really see the symptoms of sickle cell disease, including hematological profiles are quite much, much better or normal, even when you compare them with their counterparts, children without sickle cell disease. But as fetal hemoglobin declines, you start to see now the symptoms of the disease, which comes with all the other complications. So you start to see an increase in white blood count, you start to see an increase in reticulocytes counts, which are all markers of hemolysis, but also inflammation, but also disease severity. And you start to see also the way that they are now vulnerable to different infections, anemia compared to their counterparts. So their Hb would start to decline and all that. And this happens really from six months of age and post. And you can clearly see, we just had a paper that came out just last week, which shows very clearly how these profiles are different between the two groups. 

And I think that’s really important when we are looking at interventions for sickle cell disease, especially for time points that are critical and interventions that may be needed to be started quite early on when the babies are born. So for example, hydroxyurea has now been given from nine months of age, which is good, but we need more and more molecules that are probably with less side effects than what we know from hydroxyurea. And because we already have seen that profile, we have investigated the profile of these babies, even from the very early life, we should be able to really inform in terms of the interventions and the time points that are critical for them.

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