Hematology Division, Stanford University Cancer Center, Stanford, CA 94305, USA.
Leuk Lymphoma. 2013 Feb;54(2):321-8. doi: 10.3109/10428194.2012.713477. Epub 2012 Nov 15.
Patients with myelodysplastic syndrome (MDS) receiving hypomethylating agents commonly develop thrombocytopenia. This double-blind study evaluated the efficacy and safety of romiplostim, a peptibody protein that increases platelets, in patients with MDS receiving decitabine. Patients received romiplostim 750 μg (n = 15) or placebo (n = 14) and decitabine. Median platelet counts at the beginning of each decitabine cycle trended lower in placebo-treated than in romiplostim-treated patients. Bleeding events occurred in 43% of placebo-treated and 27% of romiplostim-treated patients, and platelet transfusions were administered to 57% of placebo-treated and 47% of romiplostim-treated patients. Overall clinical therapeutic response was achieved by 21% of placebo-treated and 33% of romiplostim-treated patients. Treatment was generally well tolerated. Progression to acute myeloid leukemia (AML) occurred in one patient per group. Adding romiplostim to decitabine treatment is well tolerated and may be beneficial, as indicated by trends toward higher platelet counts at the beginning of each treatment cycle and lower platelet transfusion rates and percentages of patients with bleeding events.
接受低甲基化剂治疗的骨髓增生异常综合征（MDS）患者常发生血小板减少症。这项双盲研究评估了罗米司亭（一种增加血小板的肽结合蛋白）在接受地西他滨治疗的 MDS 患者中的疗效和安全性。患者接受罗米司亭 750μg（n=15）或安慰剂（n=14）加地西他滨治疗。与罗米司亭治疗组相比，安慰剂治疗组每个地西他滨周期开始时的血小板计数中位数呈下降趋势。安慰剂治疗组有 43%的患者发生出血事件，而罗米司亭治疗组有 27%的患者发生出血事件，安慰剂治疗组有 57%的患者需要输注血小板，而罗米司亭治疗组有 47%的患者需要输注血小板。安慰剂治疗组有 21%的患者和罗米司亭治疗组有 33%的患者达到总体临床治疗反应。治疗总体上耐受良好。两组各有 1 例患者进展为急性髓系白血病（AML）。在接受地西他滨治疗的基础上添加罗米司亭治疗耐受性良好，可能有益，表现为每个治疗周期开始时的血小板计数较高、血小板输注率和出血事件患者比例较低的趋势。
