There is no standard treatment to accelerate recovery from melphalan-induced thrombocytopenia in multiple myeloma (MM) patients undergoing autologous stem cell transplantation (ASCT). Romiplostim, a thrombopoietin receptor agonist, has been developed to upregulate platelet production. This study aimed to assess the efficacy and safety of romiplostim in reducing platelet transfusions post-ASCT in MM patients. This was an investigator-initiated, single-center, open-label, comparative pilot study conducted from January to December 2022. We evaluated 18 melphalan-conditioned MM patients treated with pegylated granulocyte colony-stimulating growth factor (GCSF) (6 mg subcutaneously, SC, on day 2) and romiplostim (250 mcg SC on day 3) post-ASCT (romiplostim cohort). We compared them with 56 MM patients who had undergone ASCT and received GCSF alone (conventional or retrospective cohort, enrolled from 2015 to 2021). Efficacy endpoints included the number of mean single donor platelet(SDP) and packed red blood cell (PRBC) transfusions, time to platelet and neutrophil engraftment, and safety and tolerability assessments. In the romiplostim cohort, the average number of SDP transfusions required was significantly lower than that in the conventional cohort (1.39 vs. 3.40, p = 0.0001). Platelet engraftment occurred significantly faster in the romiplostim cohort versus the conventional cohort (9.72 vs. 12.57 days, p = 0.0018). Neutrophil engraftment days and PRBC transfusion requirements were comparable between the two groups. No deaths or major safety concerns were reported. Romiplostim demonstrated an improved efficacy and a favorable safety profile compared to the conventional approach in patients undergoing ASCT.
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