Recombinant Human Thrombopoietin Accelerates the Recovery of Platelet in Patients With Lower-Risk Myelodysplastic Syndrome: A Proof-of-Concept Study

Yuan Yang, Zengwei Tang, Jiang Ji, Chen Yang, Miao Chen, Bing Han, Yuan Yang, Zengwei Tang, Jiang Ji, Chen Yang, Miao Chen, Bing Han

Abstract

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: LR-MDS patients receiving stanozolol (2 mg, t.i.d.) and supportive care alone (non-rhTPO) or additional rhTPO were enrolled in this study prospectively. rhTPO was given at 15,000 U (q.d.) for 7 days/month for at least 3 months. Patients stopped rhTPO if the platelet count was higher than 50 × 109/L or had no effects after 3 months of treatment. The overall response (OR), complete response (CR), platelet response, side effects, clone evolution, and clinical outcome were evaluated.

Result: Thirty-five patients were enrolled: 20 (57.1%) patients in the rhTPO group and 15 (42.9%) patients in the non-rhTPO group. The demographic and baseline characteristics were balanced between the two groups. Platelet response was higher at 1 and 2 months as compared with that in the non-rhTPO group (p = 0.006 and p = 0.001, respectively). Meanwhile, the rhTPO group had a shorter time to achieve a platelet transfusion-free state compared with the non-rhTPO group (p = 0.034). Hematologic response was higher at 1 and 2 months compared with that in the non-rhTPO group (p = 0.006 and p = 0.001, respectively). There was no significant difference in the overall response or complete response at 1, 2, 3, 6, and 12 months between the two groups. One patient in the rhTPO group evolved into higher-risk MDS at 9 months. No significant difference in disease progression, infection, gastrointestinal disorders, or drug-related liver/renal injuries was 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.

Clinical trial registration: https://ichgcp.net/clinical-trials-registry/NCT04324060?cond=NCT04324060&draw=2, identifier NCT04324060.

Keywords: adverse events; hematologic response; lower-risk myelodysplastic syndrome; platelet response; recombinant human thrombopoietin.

Conflict of interest statement

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Copyright © 2021 Yang, Tang, Ji, Yang, Chen and Han.

Figures

Figure 1
Figure 1
Trial profile. Seventy-four patients were screened for eligibility; 34 patients were excluded for not meeting the inclusion criteria. Forty patients were ultimately enrolled, of whom 20 were assigned to the recombinant human thrombopoietin (rhTPO) group and the other 20 to the non-rhTPO group. Five patients in the non-rhTPO group were lost within 3 months at the initial follow-up and were excluded from the final analysis. The remaining 35 patients completed the designed treatment.
Figure 2
Figure 2
Bar plots showing the therapy response rates at different time points in the recombinant human thrombopoietin (rhTPO) group and the non-rhTPO group. Platelet response (A), hemoglobin response (B), neutrophil response (C), hematologic response (D), complete response (E), and overall response (F) rates at 1, 2, 3, 6, and 12 months in the rhTPO and non-rhTPO groups.

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