Artist illustration of blood vessels

NICE recommends NHS use of innovative blood disorder treatment

Patient suffering from a severe blood disorder may soon find some relief after NICE recommended an innovative new treatment to be incorporated for routine use on the NHS.

The brand-new treatment – the first for 25 years – will help treat acute acquired thrombotic thrombocytopenic purpura (aTTP) – a condition which can cause blood clots in small blood vessels, which leads to decreased blood flow and oxygen supply to vital organs such as the brain, heart and kidneys.

If untreated, acute aTTP can prove fatal, sometimes within hours, and the longer blood vessels remain blocked the higher the risk of illness or death are in the patient.

It most commonly affects women, disproportionately affecting people of African Caribbean family origin. It can affect people of any age – though with the average patient (median) is around 40 years old.

The new treatment, which uses the drug caplacizumab with plasma exchange and immunosuppression, will be used to treat an acute episode of aTTP in adults, and in young people aged 12 years and over, who weight at least 40kg.

Meindert Boysen, Deputy Chief Executive and Director of the Centre for Health Technology Evaluation at NICE, said: “Caplacizumab is considered to be an innovative new treatment for acute aTTP, one of the first developed for this condition for more than 25 years.

“The company worked positively with NICE and NHSE/I to present all available clinical data, to fully explore any limitations in the data and to agree a commercial arrangement. This meant our committee was able to recommend caplacizumab as a valuable new treatment option across the NHS in England.

“This recommendation will have a positive impact on those experiencing an episode of acute aTTP and ensure they receive an innovative medicine which will treat their condition and reduce their time in hospital.”

It is estimated more than 100 people each year will benefit from the recommendation of this treatment being used routinely within the NHS.

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