A brand-new treatment – the first for 25 years – for a rare blood disorder has been recommended by NICE for routine use on the NHS.
Caplacizumab with plasma exchange and immunosuppression will be used to treat acute acquired thrombotic thrombocytopenic purpura (aTTP).
Caplacizumab, also known as Cablivi and manufactured by Sanofi, will be used to treat an acute episode of acquired TTP in adults, and in young people aged 12 years and over, who weigh at least 40 kg.
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.”
Acute aTTP cases are caused by reduced activity of an enzyme called ADAMTS13. It is an autoimmune condition meaning the body’s immune system attacks the enzyme stopping it working.
The condition causes 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, it can be fatal, sometimes within hours, and the longer blood vessels remain blocked the higher the risk of illness and dying.
It is most common in women and disproportionately affects people of African Caribbean family origin. It can affect people of any age, the average patient (median) is around 40 years old.
Current standard care to treat acute aTTP includes plasma exchange and immunosuppressant medicines. Evidence presented to NICE’s independent appraisal committee showed that caplacizumab, plus standard care, reduces the time it takes to bring blood platelet levels back to normal and the number of plasma exchange treatments needed. The medicine also reduces the time patients spend in hospital and intensive care.
Adding caplacizumab to current standard care will likely reduce long-term complications and death around an acute episode, but it is unclear by how much because of limited reported data. Although there were some uncertainties in the clinical evidence, there were also likely to be additional benefits of caplacizumab in reducing the use of scarce NHS resources like donated plasma or intensive care beds.
The company provided an improved commercial arrangement following the publication of draft guidance. This allowed the committee to recommend its use as the cost of caplacizumab, for the clinical benefits it is likely to provide, is now a good use of NHS resources.
NICE has recommended treatment should be started and supervised by physicians experienced in managing conditions in which blood clots form in small blood vessels.
The first dose is administered intravenously and subsequent doses of caplacizumab are given in an injection under the skin daily, for up to 30 days or longer, if necessary, until the episode of aTTP is resolved.
It is estimated more than 100 people each year will benefit from this recommendation.