Objective: To analyze the efficacy and influencing factors of cyclosporine (CsA) alone in the treatment of children with acquired aplastic anemia (AA).
Methods: The clinical data of children diagnosed with AA and treated with CsA alone from January 1, 2016 to December 31, 2020 in the Children's Hospital of Chongqing Medical University were collected, and the efficacy and influencing factors of CsA treatment were evaluated.
Results: Among the 119 patients, there were 62 male and 57 female, with a median age of 7 years and 1 month. There were 45 cases of very severe AA (VSAA), 47 cases of severe AA (SAA), and 27 cases of non-severe AA (NSAA). At 6 months after treatment, the efficacy of VSAA was lower than that of SAA and NSAA, and there was a statistical difference (P < 0.01). 6 cases died early, 16 cases relapsed, 2 cases progressed to AML and ALL. The results of univariate analysis showed that the high proportion of lymphocyte in the bone marrow at 6 months was an adverse factor for the efficacy of CsA, while high PLT count was a protective factor (P =0.008, P =0.002). The ROC curve showed that the cut-off values of PLT count and the proportion of bone marrow lymphocyte at 6 months were 16.5×109 /L, 68.5%, respectively. Multivariate analysis showed that the high proportion of lymphocyte in bone marrow at 6 months was an independent adverse factor for IST (P =0.020, OR =0.062), and high PLT count was a protective factor (P =0.044, OR =1.038). At 3 months of treatment, CsA response and NSAA were the risk factor for recurrence (P =0.001, 0.031).
Conclusion: The efficacy of NSAA was higher than that of SAA and VSAA after 6 months of treatment with CsA alone. A high PLT count at the initial diagnosis was a good factor for the effectiveness of CsA, and a high proportion of bone marrow lymphocyte was an unfavorable factor. CsA response at 3 months and NSAA were risk factors for recurrence.
题目: 单用环孢素治疗儿童获得性再生障碍性贫血的疗效及影响因素分析.
目的: 分析单用环孢素（CsA）治疗儿童获得性再生障碍性贫血（AA）的疗效及影响因素。.
方法: 收集重庆医科大学附属儿童医院2016年1月1日至2020年12月31日确诊AA并单用CsA治疗患儿的临床资料，评价CsA治疗的疗效及影响因素。.
结果: 119例患儿中男62例，女57例，中位年龄7岁1月。极重型AA（VSAA）45例，重型AA（SAA）47例，非重型AA（NSAA）27例。治疗后6个月时VSAA患儿的疗效明显差于SAA及NSAA（P < 0.01）。早期死亡6例；16例复发；2例分别进展为AML和ALL。单因素分析结果显示，6个月时的骨髓高淋巴细胞比例对CsA疗效是不良因素，而高PLT计数则是保护因素（P =0.008，P =0.002）。ROC曲线显示，6个月时PLT计数为16.5×109/L、骨髓淋巴细胞比例为68.5%， 可以作为判断CsA疗效的临界值。多因素分析结果显示，6个月时骨髓高淋巴细胞比例是独立的CsA不良因素（P =0.020，OR =0.062），高PLT计数是保护因素（P =0.044，OR =1.038）。治疗3个月时CsA有效及NSAA是复发的危险因素（P =0.001，P ＝0.031）。.
结论: 单用CsA治疗6个月后NSAA的疗效高于SAA及VSAA。初诊时高PLT计数是CsA治疗有效的良好因素，高骨髓淋巴细胞比例是不良因素。3月时CsA有效及NSAA是复发的危险因素。.
Keywords: aplastic anemia; children; cyclosporine; influencing factors.