Treating thrombocytopenia in myelodysplastic syndromes (MDS) with romiplostim monotherapy may not increase the risk of developing acute myeloid leukemia (AML), according to a study published in The Lancet Haematology.1

Previous studies showed that romiplostim for thrombocytopenia in MDS improved hematologic outcomes (eg, decreased platelet infusions) but may increase the risk of AML.

For this double-blind phase 2 study ( Identifier: NCT00614523), investigators randomly assigned 250 patients with thrombocytopenia in MDS to receive subcutaneous romiplostim 750 μg or placebo once weekly for 58 weeks; patients were stratified by baseline platelet count and risk levels.

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Eighty-three vs 167 patients received placebo vs romiplostim, respectively. All patients in the placebo arm and 139 patients in the romiplostim arm were included in this 5-year follow-up.

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Twenty (12%) of the 167 patients in the romiplostim arm developed AML by the end of the follow-up compared with 9 (11%) of the 83 patients in the placebo arm (hazard ratio [HR], 1.06; 95% CI, 0.48-2.33; P = .88).

The mortality rate did not differ significantly between the 2 groups, with 93 (56%) deaths in the romiplostim arm vs 54 (54%) deaths in the placebo arm (HR, 1.03; 95% CI, 0.72-1.47; P = .89).

The authors concluded that “despite initial concerns about increased [AML] transformation in patients treated with romiplostim, long-term follow-up showed that treatment with romiplostim had no significant negative impact on [AML] transformation or survival.”


  1. Kantarjian HM, Fenaux P, Sekeres MA, et al. Long-term follow-up for up to 5 years on the risk of leukaemic progression in thrombocytopenic patients with lower-risk myelodysplastic syndromes treated with romiplostim or placebo in a randomised double-blind trial. Lancet Haematol. 2018 Jan 25. doi: 10.1016/S2352-3026(18)30017-6 [Epub ahead of print]