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Updated: May 19, 2026
Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome
Published on: October 3, 2018
A randomized controlled trial of romiplostim in patients with low- or intermediate-risk myelodysplastic syndrome
Peter L Greenberg1, Guillermo Garcia-Manero, Michael Moore
1Hematology Division, Stanford University Cancer Center, Stanford, CA 94305, USA. peterg@stanford.edu
Abstract:
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.
Insights
Adding romiplostim to decitabine treatment for myelodysplastic syndrome (MDS) showed potential benefits. Patients experienced higher platelet counts and fewer bleeding events and transfusions, with good tolerability.
Area of Science:
- Hematology
- Oncology
- Pharmacology
Background:
- Myelodysplastic syndromes (MDS) are a group of clonal hematopoietic stem cell disorders.
- Thrombocytopenia is a common complication in MDS patients treated with hypomethylating agents like decitabine.
- Romilostim is a thrombopoiesis-stimulating agent designed to increase platelet counts.
Purpose of the Study:
- To evaluate the efficacy and safety of romiplostim in MDS patients receiving decitabine.
- To assess the impact of romiplostim on platelet counts, bleeding events, and transfusion requirements.
- To determine the overall clinical therapeutic response and safety profile of the combination therapy.
Main Methods:
- A double-blind, placebo-controlled study was conducted.
- Patients with MDS received either romiplostim (750 μg) or a placebo, in addition to decitabine.
- Platelet counts, bleeding events, platelet transfusions, and clinical response were monitored throughout the study.
Main Results:
- Median platelet counts at the start of decitabine cycles trended higher in the romiplostim group compared to placebo.
- Fewer bleeding events (27% vs. 43%) and a lower rate of platelet transfusions (47% vs. 57%) were observed in patients receiving romiplostim.
- A higher overall clinical therapeutic response rate was seen in the romiplostim group (33% vs. 21%).
Conclusions:
- Combination therapy with romiplostim and decitabine is well-tolerated in MDS patients.
- Romilostim may offer benefits by improving platelet counts and reducing bleeding and transfusion needs.
- Further investigation into romilostim's role in managing thrombocytopenia in MDS is warranted.
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