Key clinical point: Initial concerns appear to be unfounded about romiplostim increasing risk of acute myeloid leukemia or death in thrombocytopenic patients with low-risk myelodysplasia.
Major finding: After 5 years of follow-up, similar proportions of patients in both groups developed acute myeloid leukemia (20 [12%] of 167 in the romiplostim group vs nine [11%] of 83 in the placebo group; HR 1·06 [95% CI 0·48–2·33]; p=0·88) and similar proportions had died (93 [56%] vs 54 [54%]; HR 1·03 [0·72–1·47]; p=0·89).
Study details: 5-year follow-up results from a phase 2, multicenter, double-blind trial of romiplostim treatment in patients with lower-risk myelodysplastic syndromes recruited at 109 centers in North America, Europe, Russia, and Australia. Study subjects had platelets of 20 × 109 per L or less with or without a history of bleeding or 50 × 109 platelets per L or less with a history of bleeding.
Disclosure: The study was sponsored by Amgen, the maker of romiplostim.
Source: Kantarjian H et al. The Lancet Haematology March 2018, Pages e117-e126 https://doi.org/10.1016/S2352-3026(18)30016-4
Kantarjian H et al. The Lancet Haematology March 2018, Pages e117-e126 https://doi.org/10.1016/S2352-3026(18)30016-4