General Updates | Selecting between treatment options for patients with sickle cell disease in the UK

It’s currently quite an exciting time for sickle cell disease. We have some new therapies that have come along in the last few years. The baseline management for sickle cell disease is, of course, always health education, but as I mentioned, there is penicillin prophylaxis, there is the vaccination program for sickle cell disease patients, and we also know that the only approved drug for sickle cell disease modification currently is hydroxycarbamide. We use that as a blanket for children with homozygous sickle cell disease and hemoglobin S beta 0 thalassemia. As we know these genotypes are the more severe genotypes. We start them early at the age of nine months, if they have parental buy-in, and we can also offer them to other genotypes like hemoglobin SC or hemoglobin S beta plus thalassemia if they have a more severe phenotype in the way of recurrent crisis or acute chest syndrome or other complications. So hydroxycarbamide remains for the last 40 plus years as the main drug that we use in sickle cell disease. 

We also have chronic transfusion therapy that we can offer to patients and these are selected patients that have worsening disease severity despite hydroxycarbamide or for the primary or secondary prevention of sickle cell related stroke. The transfusion therapy comes with huge benefits, but also some complications, including iron overload or development of antibodies and reactions to transfusion. 

Now, there is, of course, a huge category of stem cell therapies. One of the most well-established therapies is called the hematopoietic stem cell transplantation or bone marrow transplant, and that is still considered to be the only absolute cure for sickle cell disease. And selecting a patient for that depends on the patient’s disease severity, their history of complications, and also the availability of a donor, be it a sibling donor or parent or someone else. 

So in the same category, we have stem cell gene editing therapy that is the newest addition to the different disease treatment modalities. And that is considered to be a functional cure because it serves as a stem cell therapy that does not require an allogeneic donor. It is an autologous stem cell therapy that provides a change in the patient phenotype to the extent it is considered to be a functional cure. So this is a relatively newer modality of treatment for sickle cell disease patients, and it does come with certain criteria for patient selection, again, including what type of disease they have, how many crises they have had, and if they do or do not have any donors for stem cells. 

But overall, selecting any treatment modality depends on the disease profile, on the comorbidities profile, the patient’s own age and comorbidities. And I think most importantly, what the patient’s preferences are and what the modality is going to do to their quality of life. So that, again, should be at the core of what we do for these patients. Of course, we have their best interest at heart, and what will be the most beneficial for their individual lives is what we have to use to guide us.

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