Low-dose decitabine for refractory prolonged isolated thrombocytopenia after HCT: a randomized multicenter trial
Yaqiong Tang, Jia Chen, Qifa Liu, Tiantian Chu, Tingting Pan, Jianying Liang, Xue Feng He, Feng Chen, Ting Yang, Xiao Ma, Xiaojin Wu, Shaoyan Hu, Xingyu Cao, Xiaohui Hu, Jiong Hu, Yuejun Liu, Jiaqian Qi, Yueping Shen, Changgeng Ruan, Yue Han, Depei Wu, Yaqiong Tang, Jia Chen, Qifa Liu, Tiantian Chu, Tingting Pan, Jianying Liang, Xue Feng He, Feng Chen, Ting Yang, Xiao Ma, Xiaojin Wu, Shaoyan Hu, Xingyu Cao, Xiaohui Hu, Jiong Hu, Yuejun Liu, Jiaqian Qi, Yueping Shen, Changgeng Ruan, Yue Han, Depei Wu
Abstract
Refractory prolonged isolated thrombocytopenia (RPIT) is an intractable complication after allogeneic hematopoietic cell transplantation (HCT), which often leads to poor prognosis. A clinical study was designed to validate the efficacy and safety of low-dose decitabine for RPIT after HCT and explore the related underlying mechanisms. Eligible patients were randomly allocated to receive 1 of 3 interventions: arm A, low-dose decitabine (15 mg/m2 daily IV for 3 consecutive days [days 1-3]) plus recombinant human thrombopoietin (300 U/kg daily); arm B, decitabine alone; or arm C, conventional treatment. The primary end point was the response rate of platelet recovery at day 28 after treatment. Secondary end points included megakaryocyte count 28 days after treatment and survival during additional follow-up of 24 weeks. Among the 91 evaluable patients, response rates were 66.7%, 73.3%, and 19.4% for the 3 arms, respectively (P < .001). One-year survival rates in arms A (64.4% ± 9.1%) and B (73.4% ± 8.8%) were similar (P = .662), and both were superior to that in arm C (41.0% ± 9.8%; P = .025). Megakaryocytes, endothelial cells (ECs), and cytokines relating to megakaryocyte migration and EC damage were improved in patients responding to decitabine. This study showed low-dose decitabine improved platelet recovery as well as overall survival in RPIT patients after transplantation. This trial was registered at www.clinicaltrials.gov as #NCT02487563.
Conflict of interest statement
Conflict-of-interest disclosure: The authors declare no competing financial interests.
© 2021 by The American Society of Hematology.
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Source: PubMed