[The Efficacy and Influencing Factors of Cyclosporine Alone in the Treatment of Children with Acquired Aplastic Anemia].

Q4 Medicine

中国实验血液学杂志 Pub Date : 2024-06-01 DOI:10.19746/j.cnki.issn.1009-2137.2024.03.029

Hong-Cheng Qin, Xian-Min Guan, Yan-Ni Hu, Xiao-Ying Lei, Ying Dou, Jie Yu, Xian-Hao Wen

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Abstract

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.

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[单用环孢素治疗获得性再生障碍性贫血患儿的疗效及影响因素]。

目的分析单用环孢素（CsA）治疗获得性再生障碍性贫血（AA）患儿的疗效及影响因素：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日确诊为AA并单用CsA治疗的患儿临床资料，评价CsA治疗的疗效及影响因素：119例患者中，男62例，女57例，中位年龄7岁1个月。极重度 AA（VSAA）45 例，重度 AA（SAA）47 例，非重度 AA（NSAA）27 例。治疗 6 个月后，VSAA 的疗效低于 SAA 和 NSAA，且有统计学差异（P < 0.01）。6例早期死亡，16例复发，2例进展为AML和ALL。单变量分析结果显示，6个月时骨髓中淋巴细胞比例高是CsA疗效的不利因素，而PLT计数高是保护因素（P =0.008，P =0.002）。ROC 曲线显示，6 个月时 PLT 计数和骨髓淋巴细胞比例的临界值分别为 16.5×109 /L、68.5%。多变量分析显示，6 个月时骨髓淋巴细胞比例高是 IST 的独立不利因素（P =0.020，OR =0.062），而 PLT 计数高是保护因素（P =0.044，OR =1.038）。治疗3个月后，CsA反应和非甾体抗炎药是复发的危险因素（P =0.001，0.031）：结论：单用 CsA 治疗 6 个月后，NSAA 的疗效高于 SAA 和 VSAA。初诊时 PLT 计数高是 CsA 有效的有利因素，而骨髓淋巴细胞比例高是不利因素。3 个月的 CsA 反应和非甾体抗炎药是复发的危险因素。

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