176例再生障碍性贫血患者门诊治疗真实世界研究

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目的:探索真实世界门诊治疗再生障碍性贫血(AA)患者的诊断流程、治疗及疗效。方法:评估2018年1月至2019年12月门诊治疗176例AA患者的诊断流程、治疗方案及疗效。结果:患者出现症状至首次就诊中位时间7(5~120)个月,就诊原因包括出血(52.3%)、贫血(51.7%)、感染(6.8%)。诊断流程中168例(95.5%)患者完成髂骨骨髓穿刺(骨穿),仅22例(17.1%)完善了多部位骨穿(胸骨分类);骨髓活检完成率85.1%(143/168);只有59.5%(100/168)和58.9%(99/168)完成了骨髓流式免疫分型及染色体核型分析;86例(48.5%)完成阵发性睡眠性血红蛋白尿(PNH)克隆筛查;造血祖细胞培养检查完成率最低,仅45例(26.8%)。环孢素A(CsA)联合雄激素及左旋咪唑为最常见治疗方案,总计77例(43.8%),其次为CsA联合雄激素45例(25.6%),单用CsA者24例(13.6%),单用雄激素者16例(9.1%),14例(7.9%)患者确诊后选择中药或未治疗。根据门诊复诊频率将176例患者分为复诊规律组(每年≥4次)130例(73.9%)和复诊欠规律组(每年<4次)46例(26.1%)。复诊规律组6个月有效率较高(52.5%对28.0%,n P=0.005),12个月高质量缓解率更好(40.7%对16.7%,n P=0.027),24个月复发率更低(4.4%对36.4%,n P=0.001)。n 结论:真实世界治疗的AA患者诊断流程需重视多部位骨穿检查并完成PNH克隆筛查、流式免疫分型、染色体核型分析及造血祖细胞培养检查;门诊规律复诊患者可获得高质量缓解,复发率低,推荐每年至少复诊4次。“,”Objective:To explore the diagnostic process and outcomes of patients with aplastic anemia (AA) who received outpatient treatment in a real-world setting.Methods:The diagnostic processes, treatment regimens, and outcomes of 176 patients with AA treated in outpatient centers from January 2018 to December 2019 were reviewed.Results:The median interval from the onset of symptoms to the first visit was 7 (5-120) months. Complaints during the first visit included bleeding (52.3% ) , anemia (51.7% ) , and infection (6.8% ) . For diagnosis, 168 patients (95.5% ) underwent bone marrow aspiration; however, only 22 of them (17.1% ) consented aspiration in multiple sites (sternum) . The completion rate of bone marrow biopsy was 85.1% (143/168) ; flow immunophenotype and karyotype analyses were performed on 59.5% (100/168) and 58.9% (99/168) of AA patients, respectively, and the culture of clonal forming units by bone marrow mononuclear cells was performed on 26.8% (45/168) of AA patients. The most preferred regimen was cyclosporine combined with androgen and levamisole (43.8% , 77 patients) , followed by cyclosporine combined with androgen (25.6% , 45 patients) . Cyclosporine alone was administered in 24 patients (13.6% ) and androgen alone in 16 patients (9.1% ) . Furthermore, 14 patients (7.9% ) did not consent to any drugs or only chose traditional Chinese medicine. The patients were divided according to the frequencies of follow-up: regular follow-up group (≥4 times/year, n n=130) and irregular group (<4 times/year,n n=46) . The former had a higher 6-month remission rate (52.5% n vs 28.0% , n P=0.005) , a greater high-quality remission rate in 12 months (40.7% n vs 16.7% , n P=0.027) , and a lower relapse rate in 24 months (4.4% n vs 36.4% , n P=0.001) .n Conclusion:In real-world settings, bone marrow aspiration in multiple sites should be addressed in outpatient treatment for AA diagnostic work-up, including PNH clone screening, flow immunophenotype, chromosome karyotype analysis, and culture of clonal forming units. Patients with AA who follow regular visits were more likely to achieve high-quality remission and a lower relapse rate. Visits at least four times per year are recommended for AA patients undergoing outpatient treatment.
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