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 incurable and affects 15,000 people in the UK.

And the National Institute for Health and Care Excellence said the hope of reducing health inequalities for black people, who are predominantly affected and often have poorer health to start with, made the drug worth recommending.

It called it “an innovative treatment”.

Painful episodes

The drug, crizanlizumab, made by Novartis, is injected into a vein and can be taken on its own or alongside standard treatment and regular blood transfusions.

And in a trial, patients taking the crizanlizumab had a sickle-cell crisis 1.6 times a year on average, compared with nearly three times a year normally.

These painful episodes, which can require hospital treatment and lead to other health complications, are caused by by sickle-shaped red blood cells blocking the small blood vessels .

But because the trial was small and lasted only a year, it remains unknown how long the benefits last for – and that makes it difficult to judge how cost-effective crizanlizumab is.

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

And additional data on the treatment will be collected through clinical trials.

The charity Sickle Cell Society said the new treatment brought “new hope” for people living with the world’s most common genetic blood condition.

NHS chief executive Amanda Pritchard said: “The moment that a new drug comes that is approved to be used, our job is to make sure that we can do a deal to ensure it’s affordable and get it out as quickly as possible.”

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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