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Introduction <p>Myelodysplastic syndrome (MDS) represents a heterogeneous group of myeloid neoplasms, with approximately two-thirds identified as lower-risk MDS (LR-MDS). LR-MDS with thrombocytopenia is associated with poor prognosis, a high risk of life-threatening hemorrhage, and limited treatment options.</p> Methods <p>We explored a prospective single-arm, single-center study&#xa0;on&#xa0;the&#xa0;combination&#xa0;of very-low-dose decitabine (VLD-DAC, 3.5&#xa0;mg/m<sup>2</sup>/day from days 1 to 5 of a 28-day cycle) and recombinant human thrombopoietin (rhTPO, 1.5 million units/day from days 1 to 14) for treating LR-MDS with thrombocytopenia (platelet count &lt; 50 × 10<sup>9</sup>/L). The primary endpoint was the hematologic improvement-platelet (HI-P) rate.</p> Results <p>Among 20 patients, 19 completed at least two treatment cycles. Of these, 63% (12/19) achieved HI-P within 1&#xa0;month (range, 0.7 to 3.5&#xa0;months), 57% (4/7) of patients gained platelet transfusion independence. Furthermore, 74% (14/19) demonstrated hematologic response in at least one blood cell lineage, with 86% (6/7) achieving independent of red blood cell transfusions. During treatment, no patients experienced disease progression, but later, 2 patients did, and both ultimately succumbed. The main adverse reactions encountered during treatment were neutropenia and infection. Additionally, 2 patients succumbed to complications arising from hemorrhage or severe infection.</p> Conclusion <p>Real-world results indicate that VLD-DAC combined with rhTPO ameliorates thrombocytopenia in LR-MDS, potentially enhances patient prognosis, and demonstrates a favorable safety profile.</p>

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Very-low-dose decitabine and rhTPO for thrombocytopenia in lower-risk myelodysplastic syndrome

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年4月21日

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  • Zhen Gao,
  • Fei Yang,
  • Hong Pan,
  • Lele Zhang,
  • Weiwang Li,
  • Ruonan Li,
  • Jingyu Zhao,
  • Yuechen Luo,
  • Xiao Yu,
  • Zhexiang Kuang,
  • Neng Nie,
  • Jianping Li,
  • Jinbo Huang,
  • Xin Zhao,
  • Meili Ge,
  • Yizhou Zheng,
  • Liwei Fang,
  • Jun Shi

摘要

Introduction

Myelodysplastic syndrome (MDS) represents a heterogeneous group of myeloid neoplasms, with approximately two-thirds identified as lower-risk MDS (LR-MDS). LR-MDS with thrombocytopenia is associated with poor prognosis, a high risk of life-threatening hemorrhage, and limited treatment options.

Methods

We explored a prospective single-arm, single-center study on the combination of very-low-dose decitabine (VLD-DAC, 3.5 mg/m2/day from days 1 to 5 of a 28-day cycle) and recombinant human thrombopoietin (rhTPO, 1.5 million units/day from days 1 to 14) for treating LR-MDS with thrombocytopenia (platelet count < 50 × 109/L). The primary endpoint was the hematologic improvement-platelet (HI-P) rate.

Results

Among 20 patients, 19 completed at least two treatment cycles. Of these, 63% (12/19) achieved HI-P within 1 month (range, 0.7 to 3.5 months), 57% (4/7) of patients gained platelet transfusion independence. Furthermore, 74% (14/19) demonstrated hematologic response in at least one blood cell lineage, with 86% (6/7) achieving independent of red blood cell transfusions. During treatment, no patients experienced disease progression, but later, 2 patients did, and both ultimately succumbed. The main adverse reactions encountered during treatment were neutropenia and infection. Additionally, 2 patients succumbed to complications arising from hemorrhage or severe infection.

Conclusion

Real-world results indicate that VLD-DAC combined with rhTPO ameliorates thrombocytopenia in LR-MDS, potentially enhances patient prognosis, and demonstrates a favorable safety profile.