Transformative new sickle cell treatment now available on the NHS

Transformative new sickle cell treatment now available on the NHS

10 May 2024

Whittington Health patient Jennie is one of the first to benefit

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A new drug has been cleared by the National Institute for Health and Care Excellence (NICE) that could prove transformative for many patients with sickle cell disease (SCD), including Whittington Health patient Dr Jennifer O’Connor (Jennie) from Highgate, who has “thrived” on this new treatment.

This inherited condition causes red blood cells to take on a "sickle" shape, break, and die early, which vastly reduces the amount of oxygen they can carry and means that, for some, their red blood cells are destroyed faster than they are replaced. It leaves those affected with chronic anaemia and episodes of crippling pain, called crises, which can be life-threatening and frequently require hospital care.

Over a long period, the disorder can cause severe organ damage and intense pain if damaged red blood cells block vessels and restrict oxygen supply, which can also lead to strokes and sight loss. It affects around 17,000 people in England, and is generally more common in people of Black African, Caribbean, Middle Eastern and South Asian heritage.

Treatment has typically involved blood transfusions, and some people have had reactions to these, or have not wished to have blood transfusions.

This new drug Voxelotor is the first ever to be made available to all eligible patients on the NHS, and is available in a pill form taken once a day. It could help up to 4,000 people reduce the need for blood transfusions and require fewer hospital appointments. It can also be used in conjunction with the long-established treatment hydroxycarbamide, or alone in those who are unable to take hydroxycarbamide. 

Whittington Health offered this drug as part of an Early Access to Medicines scheme, and Jennie was one of the first patients to benefit. She is of British Jamaican Heritage, was diagnosed with SCD at birth and has lost two siblings to the disease.

According to Jennie: “Sadly, life for many years had been a constant treadmill of being tired, feeling fatigued, living with both acute and chronic pain and existing with insufficient haemoglobin in my blood. I lived knowing that there were no treatment options which were suitable for me personally. 

“Regular blood transfusions could have a serious impact on my life due to my development of antibodies, which could be life threatening. 

“My doctors also felt the frustration of not being in a position to manage my ‘unmet needs’, especially as I was ambitious and holding down a professional role which I loved as an academic.

“However, in 2022, Dr Ryan Mullally, from the Whittington Hospital contacted me to inform me that I would be a suitable candidate for the Early Access Medicine Scheme (EAMS) for Voxelotor. I undertook research into this drug, which originated in the USA, before signing the consent form, understanding the commitment to regular blood tests. My only fear was that the medication, if it worked, wouldn’t be approved!

“It was only a matter of weeks before I felt a very positive impact, which was confirmed by my haemoglobin increasing from 60g/L (very anaemic) to 92g/L. Over the last 2 years, I have never experienced any side effects and my haemoglobin has stabilised at between 80 and 100. I felt increased energy, less tired, less fatigue and over time the level of chronic pain reduced.  My pain medication has reduced significantly and there have been no acute crisis episodes. My overall quality of life for the first time felt ‘normal’ and my family noticed the changes. It’s now a matter of learning to thrive with this new normal.”

Dr Mullally, consultant haematologist at Whittington Health NHS Trust, commented: “I have had the privilege to see the difference this new drug has made to a number of my patients, and I’m delighted that this is now being made available for us to prescribe to all suitable NHS patients.”

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