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

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.