For the first time in two decades, a new treatment is available on the NHS for people with sickle cell disease.
Approximately 15,000 people in the UK have the inherited blood disorder, a lifelong genetic condition which the NHS says can only be totally cured by a stem cell or bone marrow transplant but these are not done often due to the risks involved.
As a result, those with sickle cell disease, which is more common in those with African or Caribbean background, are left to live with often-debilitating pain that flares up in periods known as a sickle cell crisis and can require hospital admission for pain management and treatment, as well as possible emergency blood transfusions.
The new drug, crizanlizumab, announced on Tuesday, will be offered to over 16s and injected via transfusion drip. It works by binding to a protein in the red blood cells to prevent the restriction of oxygen supply caused by the sickle-shaped cells that block small vessels.
The National Institute for Health and Care Excellence (NICE) says clinical evidence suggests those treated with crizanlizumab have significantly fewer sickle cell crises in a year.
These crises can take days or weeks for patients to recover from. NHS chief executive Amanda Pritchard said the drug would help as many as 5,000 people in the next three years.
Kehinde Salami, 40, from Sydenham, was diagnosed with sickle cell disease in his mid-20s when he was a university student in Manchester but had struggled with symptoms since childhood. I struggled [as] a kid, waking up and going to school, with my fitness, running, he says.
As an adult he now experiences a range of issues, including fatigue, joint pain, organ pain, swelling of the feet after walking, and even on one occasion blindness.
No day is the same, he says. A [sickle cell] crisis can happen wherever blood flows in the body, so I went blind in my left eye due to a crisis and had to have surgery to correct it. My vision is at 70 per cent now.
Last year, Mr Salami, who works for the NHS, contracted Covid-19 and says he was on the verge of death before being hospitalised. It caused me severe organ shutdown, I couldnt breathe, vomiting, blackouts. Post-Covid his energy levels have diminished further. It has been a battle.
Responding to the news of the treatment, Mr Salami says it is an amazing breakthrough and he cannot wait to see the benefits, particularly for younger sickle cell warriors like his nine-year-old daughter, or babies and toddlers.
Weve been neglected when it comes to new treatments, and we needed a win, and for me this is it, he says, adding that he would like to try it himself but [doesnt] want to take priority or take the place of someone that needs it more.
Shianne-Rose Samuels, 20, from west London, was diagnosed with sickle cell disease at birth. Both her parents have the trait.
Since she was in secondary school she has been in hospital on a near-monthly basis, sometimes more regularly, with painful crises. She also had her spleen and gallbladder removed at 17 and, as a result, is now at higher risk of infections.
Ms Samuels says: Its an invisible illness, youre not in a wheelchair or anything so [people] dont really think we need assistance and when we are well, we look well my insides are deteriorating [but] you cant see that.
Although she is extremely excited about the treatment it gives me hope for the future, it really does and would put her name forward, she worries about the accessibility of transfusion-only as she has problems with cannulation.
It is also notable that crizanlizumab is the first new treatment for the illness in 20 years despite 1 in 79 babies born in the UK carrying the sickle cell trait.
Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE, said: Sickle cell disease can be a debilitating condition that has a huge impact on patients quality of life. Yet treatment for sickle cell disease has been limited for years, and there has been a lack of treatments.
Both Ms Samuels and Mr Salami suggest more could be done for the community, including awareness and support. Mr Salami says: I just think people are ignoring it, maybe because it predominantly affects black people but its not just a black disease I think people need to start taking heed.
Ms Samuels thinks people black people are often perceived as strong, but we do suffer, she adds.
Jabeer Butt, CEO of the Race Equality Foundation, says that although the treatment is life-changing, people with sickle cell continue to face patchy care across the UK, so crizanlizumab must go hand in hand with accessible support and regular screening of at-risk populations.
Mr Salami wants to use the moment to encourage black Britons to donate blood. In July, i reported, the NHS was calling for donors because of rising demand. There are currently 12,633 black and mixed black blood donors, which is about 1.5 per cent of the total donor base.
Mr Salami is currently helping to run a blood drive, partnered with Evidence Joel, mother of Richard Okorogheye who died in April 2021 and also suffered with sickle cell disease.
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Sickle cell disease patients given hope for the future with first new treatment in 20 years - iNews
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