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Caplacizumab for treating acute acquired thrombotic thrombocytopenic purpura

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1 Recommendations

1.1 Caplacizumab with plasma exchange and immunosuppression is not recommended, within its marketing authorisation, for treating an acute episode of acquired thrombotic thrombocytopenic purpura (TTP) in adults, and in young people aged 12 years and over who weigh at least 40 kg.

1.2 This recommendation is not intended to affect treatment with caplacizumab that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop. For young people, this decision should be made jointly by them, their clinician, and their parents or carers.

Why the committee made these recommendations

Standard care for an acute episode of acquired TTP includes plasma exchange and immunosuppressant medicines. Trial results show that, compared with standard care alone, caplacizumab plus standard care reduces:

  • the time it takes to bring platelet levels back to normal

  • the number of plasma exchange treatments needed

  • time in hospital and intensive care.

Adding caplacizumab may reduce the long-term complications of acquired TTP and risk of death around the time of an acute episode. But, the trial does not look at whether adding caplacizumab improves either length or quality of life over the long term. Alternative ways estimating these outcomes are not proven, so this needs confirming.

The limitations in the clinical evidence mean that the cost-effectiveness estimates for caplacizumab compared with standard care are very uncertain. However, they may be higher than the range normally considered a cost-effective use of NHS resources. So, caplacizumab is not recommended for treating acute acquired TTP.