In a major breakthrough, National Health Service (NHS) has approved the first drug for sickle cell disease in 20 years. The "revolutionary" treatment, called the Crizanlizumab, will help over 5,000 people with the condition in the next three years, NHS said in a statement. It will also slash the number of times a sickle cell disease patient needs to go to A&E by two-fifths.
"This is a historic moment for people with sickle disease who will be given their first new treatment in over two decades. This revolutionary treatment will help to save lives, allow patients to have a better quality of life and reduced trips to A and E by almost half," NHS Chief Executive Amanda Pritchard said in a statement.
The drug prepared by Novartis was tested over a limited number of patients, thus, leaving open ends for judging its longevity. Crizanlizumab treatment involves injecting the medicine into the vein alongside standard treatment and regular blood transfusions. According to the National Institute of Health and Care Excellence (NICE), clinical evidence suggests that people treated with Crizanlizumab had significantly fewer sickle cell crises in a year than those receiving other standard treatment options.
"Crizanlizumab is an innovative treatment that has shown the potential to improve hundreds of lives, and we are delighted to be able to recommend it as the first new treatment for sickle cell disease," Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE said in a statement.
All patients above 16 years of age who suffer from sickle disease will be eligible for the Crizanlizumab treatment. Also, the drug will be available to the patients "at a price that is affordable to the taxpayers," Pritchard added. "A new treatment brings new hope for people living with sickle cell disorder, the world's most common genetic blood condition," Sickle Cell Society chair Kye Gbangbola told Evening Standard. She also explained how SCD is a medical emergency and causes excruciating pain. "(This) new treatment will reduce the number of agonising pain episodes we have to endure," she added.
Sickle cell disease is the name for a group of inherited health conditions that affect red blood cells. The most serious type is called sickle cell anaemia. As per NHS, it is particularly observed in people with African or Caribbean family backgrounds.
People affectedwith sickle cell disease produce unusually shaped red blood cells that can cause problems because they do not live as long as healthy blood cells and can block blood vessels. The most common symptoms of the disease are: painful episodes called sickle cell crises, which can be very severe and last up to a week; an increased risk of serious infections; anaemia (where red blood cells cannot carry enough oxygen around the body), which can cause tiredness and shortness of breath. Sickle cell disease is caused by a gene that affects how red blood cells develop.
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NHS approves revolutionary drug for sickle cell disease; first in 20 years - Republic World
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