NICE has published its final guidance (Technology Appraisal 221) on the use of romiplostim for the treatment of chronic immune or idiopathic thrombocytopenic purpura within the NHS.

Romiplostim acts as an agonist at thrombopoietin receptors, thereby stimulating the differentiation and proliferation of megakaryocytes and increasing platelet production and platelet counts.  It is licensed in the UK for adult chronic immune (idiopathic) thrombocytopenic purpura splenectomised patients who are refractory to other treatments (e.g. corticosteroids, immunoglobulins).

NICE has recommended romiplostim for the treatment of adults with chronic immune (idiopathic) thrombocytopenia purpura, providing that the following apply:

• Their condition is refractory to standard active treatments and rescue therapies, or

• they have severe disease and a high risk of bleeding that needs frequent courses of rescue therapies, AND

• the manufacturer makes romiplostim available with the discount agreed as part of the patient access scheme

The guidance notes that romiplostim has been shown to be cost effective only for patients with severe refractory ITP.  Only a haematologist should start and supervise treatment with romiplostim.