Yang J H, Xue M J, Zhang X L, Wei Z C, Shao L L, Shi Y, Hou M
Department of Gastroenterology, Qilu Hospital of Shandong University, Jinan 250012, China.
Zhonghua Xue Ye Xue Za Zhi. 2023 Jul 14;44(7):567-571. doi: 10.3760/cma.j.issn.0253-2727.2023.07.008.
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. 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. 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 (=28) and the non-response (NR) group (=33) revealed significant differences in responses to glucocorticoids (dependent or resistant) and platelet counts before treatment ((2)=8.789, =0.003; =-2.416, =0.016). The glucocorticoid-dependent group showed higher platelet counts than the glucocorticoid-resistant group after the second and third cycles of DAC treatment (=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 (=0.042, =0.012, =0.029). A significant correlation was observed between glucocorticoid dependence and responses to DAC treatment (=9.213, 95% 1.937-43.820, =0.005) . 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.
本研究旨在评估地西他滨（DAC）的疗效，并确定影响原发性免疫性血小板减少症（ITP）患者治疗反应的因素，这些患者糖皮质激素治疗失败。回顾性分析了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（5mg·m(-2)·d(-1)×3天，静脉输注）至少三个周期、间隔3至4周的61例糖皮质激素抵抗性ITP患者的临床资料。61例患者中，男性20例，女性41例，中位年龄45岁（范围：15至81岁）。其中，43例患者为糖皮质激素依赖型（糖皮质激素依赖组），18例患者为糖皮质激素抵抗型（糖皮质激素抵抗组）。DAC治疗后，12例患者（19.67%）达到完全缓解（CR），16例患者（26.23%）出现反应（R），总反应（OR）率为45.90%（28/61）。OR组（=28）和无反应（NR）组（=33）之间的比较显示，糖皮质激素反应（依赖或抵抗）和治疗前血小板计数存在显著差异（(2)=8.789，=0.003；=-2.416，=0.016）。在DAC治疗的第二和第三个周期后，糖皮质激素依赖组的血小板计数高于糖皮质激素抵抗组（=0.032，0.024）。此外，糖皮质激素依赖组在DAC治疗的第一、第二和第三个周期后的OR率均高于糖皮质激素抵抗组（=0.042，=0.012，=0.029）。观察到糖皮质激素依赖与DAC治疗反应之间存在显著相关性（=9.213，95% 1.937至43.820，=0.005）。DAC在一部分糖皮质激素抵抗性原发性ITP患者中显示出确切疗效且不良反应轻微。糖皮质激素依赖和治疗前较高的血小板计数与对DAC治疗的良好反应相关。
