Department of Hematology, Qilu Hospital, Shandong University, Jinan, China.
Department of Hematology, Qilu Hospital (Qingdao), Shandong University, Qingdao, China.
Am J Hematol. 2019 Dec;94(12):1374-1381. doi: 10.1002/ajh.25646. Epub 2019 Oct 17.
We conducted a prospective, multicenter study to evaluate the efficacy and safety of low-dose decitabine in adult patients with refractory immune thrombocytopenia. Adult patients who did not respond to, did not tolerate, or were unwilling to undergo splenectomy, with either a baseline platelet count less than 30 × 10 /L or the presence of bleeding symptoms and further need of ITP-specific treatments, were enrolled. Patients received decitabine at 3.5 mg/m intravenously for three consecutive days per cycle, for three cycles with a four-week interval between cycles. All patients were assessed every week during the first 12 weeks and at four-week intervals thereafter. We screened 49 patients for eligibility. Four patients were excluded and 45 received decitabine. At the end of decitabine treatment, complete response was achieved in eight patients (17.78%), and partial response was achieved in 15 patients (33.33%). The median time to initial response was 28 days (range, 14-70 days). Furthermore, seven relapsed patients received decitabine retreatment and all showed platelet response, including one complete response and six partial responses. Sustained response rates at 6, 12 and 18 months were 44.44% (20/45), 31.11% (14/45) and 20.0% (9/45), respectively. For responders, immune thrombocytopenia-related symptoms, fatigue, psychological health, fear, and overall quality of life were significantly improved. Adverse events were observed in 13 (28.89%) patients. No serious adverse events were recorded. In conclusion, low dose decitabine is potentially effective and safe in the management of adults with refractory immune thrombocytopenia. This trial is registered with clinicaltrials.gov identifier: NCT01568333.
我们进行了一项前瞻性、多中心研究，以评估小剂量地西他滨治疗成人难治性免疫性血小板减少症的疗效和安全性。纳入标准为：不符合脾切除术条件或不能耐受脾切除术，或不愿行脾切除术，基线血小板计数<30×10 /L 或存在出血症状且需要进一步接受 ITP 特异性治疗的成人患者。患者每 3 周为一个周期，接受为期 3 天的 3.5mg/m 地西他滨静脉滴注治疗，每个周期之间间隔 4 周。所有患者在前 12 周内每周评估一次，此后每 4 周评估一次。我们共筛选了 49 例患者，其中 4 例被排除，45 例接受了地西他滨治疗。地西他滨治疗结束时，8 例（17.78%）患者达到完全缓解，15 例（33.33%）患者达到部分缓解。首次反应的中位时间为 28 天（范围 14-70 天）。此外，7 例复发患者接受了地西他滨再治疗，所有患者均显示血小板反应，包括 1 例完全缓解和 6 例部分缓解。6、12 和 18 个月的持续缓解率分别为 44.44%（20/45）、31.11%（14/45）和 20.0%（9/45）。对于有反应者，免疫性血小板减少症相关症状、疲劳、心理健康、恐惧和整体生活质量均显著改善。13 例（28.89%）患者出现不良反应。无严重不良事件发生。总之，小剂量地西他滨治疗成人难治性免疫性血小板减少症具有潜在疗效和安全性。该研究已在 clinicaltrials.gov 注册，编号为：NCT01568333。
