地西他滨在糖皮质激素治疗失败原发免疫性血小板减少症患者中的疗效及影响因素

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.