Department of Blood Rheumatism Immunology, The Second Affiliated Hospital of Xiamen Medical College, Xiamen, People's Republic of China.
Department of Physical Examination, The Second Affiliated Hospital of Xiamen Medical College, Xiamen, People's Republic of China.
Hematology. 2023 Dec;28(1):2196864. doi: 10.1080/16078454.2023.2196864.
Antinuclear antibody (ANA)-positive immune thrombocytopenia (ITP) patients have an unsatisfactory prognosis due to the more severe conditions of these patients and poor response to first-line glucocorticoids (GCs). The current study intended to compare the efficacy and safety of AZA plus prednisone and prednisone alone as first-line treatment in ANA-positive ITP patients.
Fifteen ANA-positive ITP patients receiving AZA plus prednisone (AZA + GC group) and eighteen ANA-positive ITP patients receiving prednisone alone (GC group) as first-line treatment were retrospectively enrolled.
The complete response (CR) rate (60.0% versus 22.2%) (= 0.038) was increased in the AZA + GC group versus the GC group, while the overall response rate (86.7% versus 55.6%) (= 0.070) only showed an increasing trend that did not achieve statistical significance. In addition, multivariate analysis revealed that AZA + GC (versus GC) (odds ratio = 31.331, = 0.018) was independently associated with a higher possibility of achieving CR. Additionally, accumulating relapse-free duration was prolonged in the AZA + GC group versus the GC group (median: 7.8 months versus 3.4 months) (= 0.038). Additionally, the multivariate analysis suggested that AZA + GC (versus GC) (hazard ratio = 0.306, = 0.007) was independently correlated with longer accumulating relapse-free duration. The incidence of adverse events did not differ between the two groups (all > 0.05), and the common adverse events in the AZA + GC group were pneumonia (13.3%), anemia (13.3%), cough (13.3%), nausea (6.7%), and granulocytopenia (6.7%), which were all tolerable and manageable.
First-line AZA plus prednisone realizes a better hematological response and relapse-free duration with acceptable adverse events compared to prednisone alone in ANA-positive ITP patients.
抗核抗体（ANA）阳性免疫性血小板减少症（ITP）患者由于病情更严重且对一线糖皮质激素（GC）反应不佳，预后较差。本研究旨在比较 AZA 联合泼尼松与单独泼尼松作为 ANA 阳性 ITP 患者一线治疗的疗效和安全性。
回顾性纳入 15 例接受 AZA 联合泼尼松（AZA+GC 组）和 18 例接受单独泼尼松（GC 组）作为一线治疗的 ANA 阳性 ITP 患者。
AZA+GC 组完全缓解（CR）率（60.0%对 22.2%）（=0.038）高于 GC 组，而总体缓解率（86.7%对 55.6%）（=0.070）仅呈增加趋势，但未达到统计学意义。此外，多变量分析显示，与 GC 组相比，AZA+GC（比值比=31.331，=0.018）与更高的 CR 可能性相关。此外，与 GC 组相比，AZA+GC 组的无复发生存时间延长（中位数：7.8 个月对 3.4 个月）（=0.038）。此外，多变量分析表明，与 GC 组相比，AZA+GC（风险比=0.306，=0.007）与更长的累积无复发生存时间相关。两组不良事件发生率无差异（均>0.05），AZA+GC 组常见不良事件为肺炎（13.3%）、贫血（13.3%）、咳嗽（13.3%）、恶心（6.7%）和粒细胞减少（6.7%），均可耐受且可管理。
与单独使用泼尼松相比，一线 AZA 联合泼尼松在 ANA 阳性 ITP 患者中可实现更好的血液学反应和无复发生存期，且不良事件可接受。
