NICE Recommends Innovative Treatment for Severe Blood Disorder for NHS Use

Priscilla Lynch 

November 16, 2020

A brand-new treatment – one of the first for 25 years – for acute acquired thrombotic thrombocytopenic purpura (aTTP) has been recommended by the National Institute for Health and Care Excellence (NICE) for routine use in the NHS.

Caplacizumab (Cablivi, Sanofi Genzyme) with plasma exchange and immunosuppression will be used to treat an acute episode of aTTP in adults, and in young people aged ≥12 years, who weigh at least 40 kg.

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 a subcutaneous injection daily, for up to 30 days or longer, if necessary, until the episode of aTTP is resolved.

It is estimated more than 100 people annually will benefit from this recommendation.

Acute aTTP cases are caused by reduced activity of the enzyme ADAMTS13. 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 median patient age is around 40 years.

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, said NICE.

This article originally appeared on Univadis, part of the Medscape Professional Network.


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