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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

The Lancet Haematology Feb 01, 2018

Kantarjian HM, et al. - In order to evaluate the risk of leukaemic progression in thrombocytopenic patients with lower-risk myelodysplastic syndromes treated with romiplostim or placebo in a phase 2, multicentre, double-blind trial, researchers performed a 5-year follow-up in those patients. Following the decision to stop the study drug, no difference was apparent in 5-year long-term follow-up hazard ratios (HRs) for transformation to acute myeloid leukaemia and HRs for death between patients treated with romiplostim and those treated with placebo. This finding reflects that despite initial concerns, use of romiplostim was probably not associated with any increased risk of acute myeloid leukaemia or death.
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