Aim: The effect of recombinant human thrombopoietin (rhTPO) is largely unknown in lower-risk myelodysplastic syndrome (LR-MDS). This study aimed at investigating the safety and efficacy of rhTPO in patients with LR-MDS.

Methods: This was a prospective study. Patients newly diagnosed as LR-MDS patients from 2018 to 2020 were enrolled and randomized into two groups. They were either treated with rhTPO+ stanozolol 2mgtid (rhTPO group) or stanozolol 2mgtid alone (non-rhTPO). rhTPO was given at 15000U qd for 7days/months for at least three months. Patients stopped rhTPO if platelet count was higher than50×10 9/L, or had no effects after 3-month treatment. The overall response (OR), complete response (CR), platelet response, side effects, clone evolution and clinical outcome were evaluated.

Result: 35 patients were enrolled, the median age (range) was 55 (41-91) years and 17 patients were males, 18 patients were females. 20 (57.1%) patients were enrolled in rhTPO group and 15 (42.9%) patients in non-rhTPO group. The demographic and baseline characteristic were balanced between the two groups. The platelet response was higher at 1 month and 2 months, as compared with that in non-rhTPO group (40% vs 0%, p = 0.006; 55.0% vs0%, p = 0.001, respectively). Meanwhile, rhTPO group had a shorter time to achieve platelet transfusion free compared with non-rhTPO group (2 (1-6) months vs 4.5 (2-12) months, p=0.034). No significant difference in overall response, complete response, or hematologic response at 1/2/3/6/12months between the two groups. One patient in rhTPO group evolved into higher risk MDS, one patient died of intracranial hemorrhage. No significant difference in disease progression, infection, gastrointestinal disorders or drug-related liver/renal injuries were found between the two groups (p>0.05).

Conclusion: Adding short-term rhTPO can accelerate the early platelet response and decrease platelet transfusion with no obvious side effects.

Disclosures

No relevant conflicts of interest to declare.

© 2021 by The American Society of Hematology
2021
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