Fostamatinib for treating refractory chronic immune thrombocytopenia
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1 Recommendations
1.1 Fostamatinib is not recommended, within its marketing authorisation, for treating refractory chronic immune thrombocytopenia in adults.
1.2 This recommendation is not intended to affect treatment with fostamatinib 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.
Why the committee made these recommendations
Treatment for refractory chronic immune thrombocytopenia after thrombopoietin receptor agonists or when they are not suitable includes rituximab or mycophenolate. Fostamatinib would be used after thrombopoietin receptor agonists or when they are not suitable.
Clinical evidence shows that fostamatinib is moderately effective compared with placebo. There is no clinical trial evidence directly comparing fostamatinib with rituximab or mycophenolate. The company chose rituximab with 'watch and rescue' (monitoring until a bleed happens and treatment is needed) as its comparator. However, it did not provide any direct or indirect evidence comparing rituximab with fostamatinib.
This means that the cost-effectiveness estimates for fostamatinib are very uncertain. They are likely to be higher than what NICE normally considers cost effective. So, fostamatinib is not recommended.
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