Fostamatinib for treating refractory chronic immune thrombocytopenia

1.1 Fostamatinib is recommended as an option for treating refractory chronic immune thrombocytopenia (ITP) in adults, only if:

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 options for refractory chronic ITP include TPO‑RAs, which are mostly followed by rituximab or mycophenolate. Fostamatinib is licensed for treating refractory chronic ITP, but the company has only provided evidence for using fostamatinib after a TPO‑RA, or when they are not suitable. Fostamatinib would be used at the same point in the treatment pathway as rituximab or mycophenolate.

Clinical evidence shows that fostamatinib is effective compared with placebo. There is no clinical trial evidence directly comparing fostamatinib with rituximab or mycophenolate. An indirect comparison shows that fostamatinib works better than rituximab at increasing the number of platelets in the blood (cells that help the blood to clot).

The cost-effectiveness estimates for fostamatinib compared with rituximab are within what NICE normally considers an acceptable use of NHS resources. So, fostamatinib is recommended.