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Sunday, October 24, 2021

First new treatment for sickle cell in 20 years


The first new sickle-cell treatment in 20 years will help keep thousands of people out of hospital over the next three years, NHS England has said.

Sickle-cell disease is an incurable disease that affects 15,000 people in the United Kingdom.

According to the National Institute for Health and Care Excellence, the drug is worth recommending because it has the potential to reduce health inequalities for black people, who are disproportionately affected and often have poorer health to begin with.

It was described as “an innovative treatment.”


Painful episodes

The Novartis drug, crizanlizumab, is injected into a vein and can be used alone or in conjunction with standard treatment and regular blood transfusions.

In addition, patients taking crizanlizumab experienced a sickle-cell crisis 1.6 times per year on average, compared to nearly three times per year normally.

These painful episodes, which can necessitate hospitalisation and result in other health complications, are caused by sickle-shaped red blood cells that block small blood vessels.

However, because the trial was small and only lasted a year, it is unclear how long the benefits will last, making it difficult to determine how cost-effective crizanlizumab is.

Nonetheless, NICE, which recommends treatments in England and Wales, recommends its use for over-16s on the NHS, albeit under a special arrangement rather than routinely.

In addition, additional information about the treatment will be gathered through clinical trials.

According to the Sickle Cell Society, the new treatment provides “new hope” for people suffering from the world’s most common genetic blood condition.

‘Every day is pain’

Sarah-Jane has to rely on a whole range of medication to manage her sickle-cell disease, including very strong painkillers

Sarah-Jane Nkrumah, 27, was diagnosed at birth and had her first crisis at the age of six months. She now suffers from chronic joint pain.

“Every day is a struggle,” she says.

“I can’t remember the last time I was completely pain-free.”

Sarah-Jane prefers to take painkiller breaks, but some days she can’t get out of bed.

“You just have to try to deal with it,” she says.

“I don’t have much of a choice.

“It all boils down to having a lot of mental fortitude and support.”

She also gets a blood transfusion every six weeks to help her energy levels.

“I feel weak and exhausted before them, but refreshed and stronger afterwards,” she says.

“Thanks to the generosity of donors, I have the opportunity to live another day.”

Sarah-Jane had to abandon her dream of becoming a nursery teacher because it put her in danger of contracting a serious infection.

“Now that I’ve discovered my true calling, I enjoy raising awareness about sickle cell disease,” she says.

NICE’s deputy chief executive and director of the Centre for Health Technology Evaluation, Meindert Boysen, stated: “For years, treatment for sickle cell disease has been limited, and there has been a lack of treatments for patients whose lives have been impacted by the condition.

“Crizanlizumab… has demonstrated the potential to improve hundreds of lives, and we are pleased to be able to recommend it as the first new treatment for sickle cell disease in two decades.”

What is sickle cell disease?

  • 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
  • A simple blood test will show whether someone has it
  • Children with sickle cell are at greater risk of stroke
  • Other symptoms can include serious infections, anaemia and tiredness

Source: Sickle Cell Society / NHS UK

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