Phase 2 study of romiplostim in patients with low-or intermediate-risk myelodysplastic syndrome receiving azacitidine therapy

HM Kantarjian, FJ Giles, PL Greenberg… - Blood, The Journal …, 2010 - ashpublications.org
HM Kantarjian, FJ Giles, PL Greenberg, RL Paquette, ES Wang, JL Gabrilove…
Blood, The Journal of the American Society of Hematology, 2010ashpublications.org
We evaluated the efficacy and safety of romiplostim, a thrombopoietin mimetic, in patients
with low-or intermediate-risk myelodysplastic syndromes (MDS) receiving azacitidine
therapy. Forty patients with low-or intermediate-risk MDS were stratified by baseline platelet
counts (< 50 vs≥ 50× 109/L) and randomized to romiplostim 500 μg or 750 μg or placebo
subcutaneously once weekly during 4 cycles of azacitidine. The primary endpoint was the
incidence of clinically significant thrombocytopenic events, defined by grade 3 or 4 …
Abstract
We evaluated the efficacy and safety of romiplostim, a thrombopoietin mimetic, in patients with low- or intermediate-risk myelodysplastic syndromes (MDS) receiving azacitidine therapy. Forty patients with low- or intermediate-risk MDS were stratified by baseline platelet counts (< 50 vs ≥ 50 × 109/L) and randomized to romiplostim 500 μg or 750 μg or placebo subcutaneously once weekly during 4 cycles of azacitidine. The primary endpoint was the incidence of clinically significant thrombocytopenic events, defined by grade 3 or 4 thrombocytopenia starting on day 15 of the first cycle or platelet transfusion at any time during the 4-cycle treatment period. No formal hypothesis testing was planned. The incidence of clinically significant thrombocytopenic events in patients receiving romiplostim 500 μg, romiplostim 750 μg, or placebo was 62%, 71%, and 85%, respectively. The incidence of platelet transfusions was 46%, 36%, and 69%, respectively. These differences were not statistically significant with the small numbers in each group. Romiplostim 750 μg significantly raised median platelet counts during cycle 3 on day 1 (P = .0373) and at the nadir (P = .0035) compared with placebo. Grade 3 rash and arthralgia each were reported in 1 romiplostim-treated patient (4%). This study suggests romiplostim may provide clinical benefits in MDS patients during azacitidine therapy. This study was registered at www.clinicaltrials.gov as #NCT00321711.
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