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

Fecha de publicación: 25 January 2018

DOI: https://doi.org/10.1016/S2352-3026(18)30016-4

Autores: Hagop M Kantarjian, Pierre Fenaux, Mikkael A Sekeres, Jeffrey Szer, Uwe Platzbecker, Andrea Kuendgen, Gianluca Gaidano, Wieslaw Wiktor-Jedrzejczak, Nancy Carpenter, Bhakti Mehta, Janet Franklin, Aristoteles Giagounidis

Background: Treatment options for thrombocytopenia in myelodysplastic syndromes are scarce. As described previously in a randomised phase 2 study (n=250), 58 weeks of romiplostim treatment in patients with International Prognostic Scoring System (IPSS)-defined lower-risk (low-risk or intermediate-1 risk) myelodysplastic syndromes led to reduced platelet transfusions (p<0·0001) and increased International Working Group-defined haematological improvement–platelet rates (p<0·0001) versus placebo. 

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