Sickle cell patients ‘give hope for the future’ with first new treatment in 20 years

For the first time in two decades, a new treatment is available on the NHS for people with sickle cell disease.

Around 15,000 people in the UK suffer from an inherited blood disease, a lifelong genetic condition which the NHS says can only be fully cured with a stem cell or bone marrow transplant – but it is not often done because of the risks involved.

As a result, people with sickle cell disease, which is more common in people of African or Caribbean descent, experience the often debilitating pain that flares up during periods known as a “sickle cell crisis” and may require treatment. hospitalization for the management and treatment of pain, as well as possible emergency blood transfusions.

The new drug, crizanlizumab, announced on Tuesday, will be offered to people over 16 and injected by transfusion infusion. It works by binding to a protein in red blood cells to prevent restriction of oxygen supply caused by sickle-shaped cells that block small vessels.

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The National Institute for Health and Excellence in Care (NICE) says clinical evidence suggests that people treated with crizanlizumab have “significantly fewer sickle cell crises in one year.”

These attacks can take days or weeks for patients to recover. NHS chief executive Amanda Pritchard said the drug would help up to 5,000 people over the next three years.

Kehinde Salami, 40, from Sydenham, was diagnosed with sickle cell disease in his mid-20s while a student at Manchester University but had suffered symptoms since childhood. ” I fought [as] a kid, wake up and go to school, with my physical shape, run, ”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] a seizure can happen anywhere blood is flowing in the body, so I went blind in my left eye due to a seizure and had to have surgery to correct it. My vision is 70 percent now.

Last year Mr Salami, who works for the NHS, contracted Covid-19 and says he was “on the verge of death” before being hospitalized. “It caused me severe organ shutdown, I couldn’t breathe, vomiting, fainting.” After Covid, his energy levels declined further. “It was a battle.”

Responding to treatment announcement, Mr Salami says it is an “incredible breakthrough” and that he “can’t wait to see the benefits”, especially for “young warriors with sickle cell disease” like his daughter. nine-year-olds, or babies and toddlers. .

“We have been overlooked when it comes to new treatments, and we needed a win, and for me that’s it,” he said, adding that he would like to try it himself but “[doesn’t] want to have priority or take the place of someone who needs it most ”.

Shianne-Rose Samuels, 20, from west London, was diagnosed with sickle cell anemia at birth. Both parents have the trait.

Since she was in high school, she has been hospitalized almost every month, sometimes more regularly, with painful attacks. She also had her spleen and gallbladder removed at age 17 and, as a result, is now at higher risk for infections.

Shianne-Rose Samuels, 20, has been hospitalized almost every month with painful attacks of sickle cell disease (Photo: Shianne-Rose Samuels)

Mrs Samuels says: “It’s an invisible disease, you are not in a wheelchair or anything else [people] don’t really think we need any help and when we are well we look good … my insides are deteriorating [but] you can’t see it.

Although she is “extremely excited” by the treatment – “it gives me hope for the future, it really is” – and introduces herself, she is concerned about the accessibility of transfusion only because she has cannulation problems.

It is also noteworthy that crizanlizumab is the first new treatment for the disease in 20 years, despite the fact that one in 79 babies born in the UK carries the sickle cell trait.

Meindert Boysen, Deputy CEO and Director of the Center for Health Technology Evaluation at NICE, said: “Sickle cell disease can be a debilitating disease that has a huge impact on the quality of life of patients. Yet treatment for sickle cell disease has been limited for years and there has been a lack of treatment. “

Ms. Samuels and Mr. Salami suggest that more could be done for the community, including awareness and support. Mr Salami said: “I just think people ignore it, maybe because it mainly affects black people, but it’s not just a black disease… I think people have to start taking it into account. “.

Ms. Samuels believes black people are often seen as “strong”, “but we are suffering,” she adds.

Jabeer Butt, CEO of the Race Equality Foundation, said that although treatment is ‘life changing’, people with sickle cell disease continue to face ‘uneven care’ across the UK, so crizanlizumab must go “Hand in hand” with accessible support and regular screening of populations at risk.

Mr Salami wants to take the opportunity to encourage black Britons to donate blood. In July, I reported, the NHS was calling for donors due to growing demand. There are currently 12,633 Black and Métis blood donors, representing approximately 1.5% of the total donor base.

Mr. Salami is currently helping organize a blood drive, in partnership with Evidence Joel, mother of Richard Okorogheye who died in April 2021 and also suffers from sickle cell disease.

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