Objective: This study aimed to evaluate the efficacy of decitabine (DAC) and identify factors influencing treatment responses in patients with primary immune thrombocytopenia (ITP) who had failed glucocorticoid therapy. Methods: Clinical data of 61 patients with glucocorticoid-resistant ITP who received DAC therapy (5 mg·m(-2)·d(-1)×3 d via intravenous infusion) for at least three cycles with 3-4-week intervals at the Department of Hematology, Qilu Hospital of Shandong University, from November 2015 to June 2021 were analyzed retrospectively. Results: The 61 patients comprised 20 males and 41 females, with a median age of 45 years (range: 15-81 years). Among them, 43 patients were glucocorticoid-dependent (glucocorticoid-dependent group), while 18 patients were glucocorticoid-resistant (glucocorticoid-resistant group). Following DAC treatment, 12 patients (19.67% ) achieved complete response (CR), and 16 patients (26.23% ) exhibited response (R), resulting in an overall response (OR) rate of 45.90% (28/61). Comparison between the OR group (n=28) and the non-response (NR) group (n=33) revealed significant differences in responses to glucocorticoids (dependent or resistant) and platelet counts before treatment (χ(2)=8.789, P=0.003; z=-2.416, P=0.016). The glucocorticoid-dependent group showed higher platelet counts than the glucocorticoid-resistant group after the second and third cycles of DAC treatment (P=0.032, 0.024). Moreover, the OR rates after the first, second, and third cycles of DAC treatment in the glucocorticoid-dependent group were all higher than those in the glucocorticoid-resistant group (P=0.042, P=0.012, P=0.029). A significant correlation was observed between glucocorticoid dependence and responses to DAC treatment (OR=9.213, 95% CI 1.937-43.820, P=0.005) . Conclusion: DAC demonstrates definitive efficacy with mild adverse effects in a subset of patients with glucocorticoid-resistant primary ITP. Glucocorticoid dependence and higher platelet counts before treatment are associated with a favorable response to DAC therapy.
目的： 评估地西他滨（DAC）在糖皮质激素治疗失败原发免疫性血小板减少症（ITP）中的疗效及影响因素。 方法： 纳入2015年11月至2021年6月在山东大学齐鲁医院血液科接受DAC治疗（5 mg·m(-2)·d(-1)×3 d静脉滴注，至少应用3个疗程，每疗程间隔3~4周）的61例糖皮质激素治疗失败ITP患者，对其临床资料进行回顾性分析。 结果： 61例患者中，男20例，女41例。中位年龄45（15~81）岁。糖皮质激素依赖43例，无效18例。DAC治疗后，12例（19.67%）患者获得完全反应（CR），16例（26.23%）有效（R），总有效（OR）率为45.90%（28/61）。DAC治疗OR组（28例）与无效组（NR，33例）比较，糖皮质激素的反应性（依赖或无效）、治疗前血小板计数差异具有统计学意义（χ(2)＝8.789，P＝0.003；z＝-2.416，P＝0.016）。糖皮质激素依赖组与糖皮质激素无效组比较，DAC治疗2、3疗程后血小板计数较高（P＝0.032、0.024）；DAC治疗1、2、3疗程OR率比较，糖皮质激素依赖组均高于糖皮质激素无效组（P＝0.042，P＝0.012，P＝0.029）。糖皮质激素依赖与DAC疗效具有明显相关性（OR＝9.213，95% CI 1.937~43.820，P＝0.005）。 结论： DAC对于部分糖皮质激素治疗失败的ITP患者具有确切的疗效且不良反应轻微。糖皮质激素依赖、治疗前血小板计数高的患者DAC疗效较好。.
Keywords: Decitabine; Efficacy; Influencing Factor; Primary Immune Thrombocytopenia.