New sickle cell treatment administered to first patients in England

pharmafile | February 24, 2022 | News story | Business Services  

Sickle cell patients have now begun receiving the first new treatment for the blood disorder in over 20 years. The disease can cause severe pain and organ failure, often requiring hospital admissions.

Crizanlizumab is administered as a monthly infusion, and is anticipated to cut visits to A&E by 40%. It is a monoclonal antibody, binds to a protein on blood cells, preventing them from clumping.

Sickle cell disease causes red blood cells to distort and become sticky, blocking vessels and restricting oxygen supply, which causes excruciating pain. It causes frequent infections, swelling in the hands and legs, pain, severe tiredness, and delayed growth or puberty.

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It is inherited from both parents, who pass on a particular gene. It is possible to carry the gene without having the disease. Nearly 300 babies are born with it each year in the UK, and children with sickle cell are at greater risk of stroke. Other symptoms can include serious, infections, anaemia, and tiredness.

A report from MPs last year found “serious failings” in sickle cell care with some evidence of discrimination against patients. Dr Shivan Pancham, a consultant haematologist at Birmingham City Hospital, shared with the BBC: “Our patients often find the experience in emergency departments challenging with a lack of understanding of the severity of pain. It is hoped with these new therapies if we reduce the likelihood of attending emergency departments, ultimately this will be much better for the patients.”

Lina Adams

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