NICE has issued new guidance that recommends caplacizumab with plasma exchange and immunosuppression as an option for treating an acute episode of acquired thrombotic thrombocytopenic purpura (TTP) in adults and in young people aged ≥12 years who weigh at least 40 kg.
In announcing the decision, NICE said trial results show that, compared with standard care alone, caplacizumab plus standard care reduces the time to platelet normalisation and the number of plasma exchange treatments needed, as well as time in hospital and ICU.
The guidance says adding caplacizumab to plasma exchange and immunosuppression is likely to reduce the long-term complications of acquired TTP and decrease the risk of acquired TTP-related death, but it is unclear by how much as trial results do not address whether adding caplacizumab improves either length or quality of life in the long term after people stop taking the drug. Also, there are limited reported data on the long-term complications of acquired TTP after an acute episode.
NICE said the assumptions in the company’s economic modelling are plausible. Also, the agency said, there are potential benefits with caplacizumab that are not included in the cost-effectiveness estimates. It was concluded that the estimates are within the range normally considered a cost-effective use of NHS resources and allow NICE to recommend caplacizumab for treating acute acquired TTP.
