Improving the lives of patients with sickle cell disease: Meet Steven Okoli

Sickle cell disease is the most common genetic blood condition in the UK, affecting approximately 17,500 people. Here, Steven Okoli, consultant in red cell haematology, discusses his research into improving treatment and health outcomes for patients with sickle cell disease.

What is your role at the Trust and how long have you worked at the Trust?

I am a consultant in red cell haematology and I have worked at the Trust since April 2021. I care for patients with inherited blood disorders which affect red blood cells, such as sickle cell disease.

People with sickle cell disease do not make haemoglobin properly, a substance in red blood cells, which carry oxygen around the body. Normal red blood cells are flexible and disc-shaped, but in sickle cell disease they become rigid and shaped like a crescent or sickle because haemoglobin clumps together. These unusual cells do not live as long as normal red blood cells and can become stuck in blood vessels.

What do you love most about your role?

The work I do helps improve the lives of patients who are underrepresented and misunderstood. Sickle cell disease predominantly affects those who have an African or Caribbean family background.

Patients with sickle cell can experience excruciatingly painful episodes in any part of their body, organ damage and early death but treatment options are still very limited. I want to help improve life outcomes for the patients I care for but also work on research to find new treatments, so that all sickle patients can live happier and longer lives.

What kind of treatment is currently available for patients with sickle cell disease?

Treatment options are limited. Until recently, care for these patients was mainly to support them to live with the disease. Blood transfusions and a single medication option, which helps manage painful episodes, have been the cornerstone of treating the disease for decades. We are starting to see some innovations such as blood group matching which can reduce complications associated with transfusions and improved blood transfusion selection that ensures timely and appropriate care.

Over the coming years, new medicines which can increase haemoglobin and even potential cures such as stem cell transplants will revolutionise treatment. However, there is still much to be done in developing more and better targeted treatments.

What needs to change to improve care for patients with sickle cell?

The single most important change is to improve education. We need to provide more information for decision makers so that the necessary resources are allocated to improve care and for the pharmaceutical industry so that new treatment options can be developed. We must also work with healthcare professionals across the NHS to ensure patients with sickle cell receive the care and compassion they deserve.

We also need to support communities with education to reduce the stigma associated with the disease. This would help people with sickle cell to discuss their experiences and ask for help without judgment as well as encourage testing so that we know more about the potential for passing sickle cell on to children.

Tell us more about the research you lead and why it is so vital.

I lead research to better understand and treat sickle cell disease. I work on clinical trials to help us identify patients at risk of complications so that we can treat them appropriately and even prevent these outcomes. I am also leading studies on new medicines that could actually change the red blood cells and haemoglobin, therefore reducing symptoms.

Ultimately, we would like to see a cure for sickle cell disease accessible to all within the next decade, if not sooner. I am also leading studies at the Trust to develop treatments that could cure these patients of their disease.

Why is diversity so important when recruiting to clinical trials, especially for sickle cell disease?

Diversity in clinical research is critical. Understanding genetic disease, especially, can also help understand normal function, which benefits us all. But diversity is also vital to ensure that new treatments benefit the whole population – in the UK and globally. It’s so important that everyone is engaged and no-one feels marginalised.

We've recruited some of the first patients in the UK/Europe to sickle cell trials - how do our teams work together to make this happen?

This is only achieved through teamwork. We have over 600 adult patients with sickle cell disease at Imperial College Healthcare and we have a good relationship with our patients. They are really interested in research and we keep everyone updated on new studies, explaining the potential benefit for both the patient themselves and their community.

We also have an excellent clinical trials team at the Trust, who work closely with the medical team that means we can get a patient smoothly on to a clinical trial that may benefit them and have significant wider impact. There are a variety of community engagement and education events. We still have many challenges but our patients have developed a trust in us that means we can really support them to consider taking part in clinical trials.