儿童获得性非重型再生障碍性贫血预后影响 因素的单中心回顾性分析

Single-Center Retrospective Analysis of Prognostic Factors for Acquired Non-Severe Aplastic Anemia in Children

摘要: 目的：探讨影响环孢素A (CsA)联合雄激素治疗儿童获得性非重型再生障碍性贫血(non-severe aplastic anemia, NSAA)预后的相关因素，为优化治疗策略提供依据。方法：采用回顾性队列研究，纳入2013年1月至2023年1月收治的105例NSAA患儿，均接受CsA联合雄性激素治疗。收集临床资料，包括年龄、病程、治疗前血常规、骨髓检查以及治疗反应等。通过单因素分析(卡方检验、t检验、非参数检验)筛选潜在预后因素。结果：CsA联合雄激素方案治疗儿童NSAA总体有效率在治疗6个月、1年及2年时分别为63.81%、72.38%和66.67%；初诊时骨髓造血容积与预后呈正相关(P < 0.05)；治疗6月、1年、2年时，有效组和无效组同期中性粒细胞、血红蛋白和血小板水平比较差异均有统计学意义(P均<0.05)。结论：CsA联合雄激素治疗儿童NSAA疗效确切，发病时骨髓造血容积与疗效呈正相关，治疗无效者需警惕发展成SAA。

Abstract: Objective: To investigate the factors influencing the prognosis of cyclosporine A (CsA) combined with androgen therapy in children with acquired non-severe aplastic anemia (NSAA), and to provide evidence for optimizing treatment strategies. Methods: A retrospective cohort study was conducted. A total of 105 children with NSAA admitted from January 2013 to January 2023 were enrolled. All patients received treatment with CsA combined with androgens. Clinical data were collected, including age, disease duration, pretreatment blood routine examination, bone marrow examination, and treatment response. Potential prognostic factors were screened using univariate analysis (chi-square test, t-test, non-parametric test). Results: The overall response rates of CsA combined with androgen regimen in the treatment of pediatric NSAA were 63.81%, 72.38%, and 66.67% at 6 months, 1 year, and 2 years of treatment, respectively. Bone marrow hematopoietic volume at initial diagnosis was positively correlated with prognosis (P < 0.05). At 6 months, 1 year, and 2 years of treatment, there were statistically significant differences in the levels of neutrophils, hemoglobin, and platelets between the effective group and the ineffective group during the same period (all P < 0.05). Conclusions: CsA combined with androgen is effective in the treatment of pediatric NSAA. Bone marrow hematopoietic volume at disease onset is positively correlated with treatment efficacy. Patients who do not respond to treatment should be monitored for progression to severe aplastic anemia (SAA).