Amgen have issued a Direct Healthcare Professional Communication regarding the risk of disease progression to acute myelogenous leukaemia (AML) when using romiplostim (Nplate®) in the treatment of patients with myelodysplastic syndrome (MDS).

Romiplostim is currently licensed for the treatment of adult chronic immune (idiopathic) thrombocytopenic purpura (ITP) in splenectomised patients who are refractory to other treatments (e.g. corticosteroids, immunoglobulins), and may be considered as second line treatment for adult non-splenectomised patients where surgery is contra-indicated. 

An independent data monitoring committee recommended the termination of a clinical study evaluating the use of romiplostim in patients with MDS and thrombocytopenia (an unlicensed indication) in February 2011, due to an observed increase in the risk of progression to AML (2 patients in the placebo arm versus 9 in the romiplostim arm; percentages are not stated but the randomisation was 2:1).  There were also more patients in the romiplostim arm with increases in circulating blasts >10% (3 versus 25, respectively).

In view of the data from this study and in agreement with the MHRA, Amgen are advising healthcare professionals that romiplostim should not be used for the treatment of thrombocytopenia due to MDS or any other cause of thrombocytopenia other than ITP (i.e. as per the license), outside of clinical trials. The diagnosis of ITP should have been confirmed by the exclusion of other clinical entities presenting with thrombocytopenia, and the diagnosis of MDS must be excluded (normally by carrying out a bone marrow aspirate and biopsy) before initiating treatment with romiplostim. 

The product information for Nplate® has been updated with information on the increased risk of progression to AML in MDS patients.  Please see the link below for further details.