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中国 301 例再生障碍性贫血患者的治疗结果:10 年随访及单中心真实世界数据

Treatment outcome of 301 aplastic anemia patients in China: a 10-year follow-up and real-world data from single institute experience.

机构信息

Department of Hematology, Affiliated Hospital of Xuzhou Medical University, Xuzhou, People's Republic of China.

Institute of Hematology, Xuzhou Medical University, Xuzhou, People's Republic of China.

出版信息

Hematology. 2021 Dec;26(1):1025-1030. doi: 10.1080/16078454.2021.2009646.

DOI:10.1080/16078454.2021.2009646
PMID:34895103
Abstract

OBJECTIVE

This study was carried out to explore clinical treatment and prognosis of patients with AA with different economic status. Methods: We retrospectively analyzed the clinical outcome of 301 patients with AA in our center from April 2008 to November 2017.

RESULTS

Treatments included anti-thymocyte globulin (ATG) or anti-lymphocyte globulin (ALG) combined with cyclosporineA (CsA) (9%), allogeneic hematopoietic stem cell transplantation (allo-HSCT) (7%), CsA combined with androgen or CsA alone (hereinafter referred to as CsA group) (77%), no specific therapy (7%). The 5-year overall survival (OS) was higher in patients with non-severe AA (94.6%) compared with those with severe AA (SAA) (66.6%, <.001), very severe AA (VSAA) (41.3%, <.001). The 5-year OS was 76.5% in patients with SAA/VSAA treated with ATG/ALG combined with CsA, 75% in allo-HSCT group(P =.936), 63.6% in CsA group ( =.557), which was significantly higher than no specific therapy group (21.8%, =.002). For those who responded to CsA , the duration of CsA (median follow-up time: 27 months, 1-101 months) was positively correlated with progression-free survival (r=0.603, <.001). Multivariate analysis revealed that 36-65 years of age, SAA/VSAA, and no specific therapy were independent risk factors for inferior survival.

CONCLUSION

The treatment of elderly patients with AA still faces challenges. CsA is benefit to the survival of SAA/VSAA patients. AA patients, who responded to initialy CsA treatment, may benefit from prolonged CsA treatment. In view of the side effects of CsA, the timing of withdrawal is worth further exploration.

摘要

目的

本研究旨在探讨不同经济状况的再生障碍性贫血(AA)患者的临床治疗和预后。方法:我们回顾性分析了 2008 年 4 月至 2017 年 11 月我院收治的 301 例 AA 患者的临床转归。

结果

治疗方法包括抗胸腺细胞球蛋白(ATG)或抗淋巴细胞球蛋白(ALG)联合环孢素 A(CsA)(9%)、异基因造血干细胞移植(allo-HSCT)(7%)、CsA 联合雄激素或单独 CsA(以下简称 CsA 组)(77%)、无特殊治疗(7%)。非重型 AA(94.6%)患者的 5 年总生存(OS)明显高于重型 AA(SAA)(66.6%,<.001)和极重型 AA(VSAA)(41.3%,<.001)。SAA/VSAA 患者采用 ATG/ALG 联合 CsA 治疗的 5 年 OS 为 76.5%,allo-HSCT 组为 75%(P=.936),CsA 组为 63.6%(P=.557),明显高于无特殊治疗组(21.8%,<.001)。对于对 CsA 有反应的患者,CsA 的持续时间(中位随访时间:27 个月,1-101 个月)与无进展生存呈正相关(r=0.603,<.001)。多因素分析显示,年龄 36-65 岁、SAA/VSAA 和无特殊治疗是生存不良的独立危险因素。

结论

老年 AA 患者的治疗仍面临挑战。CsA 有利于 SAA/VSAA 患者的生存。对初始 CsA 治疗有反应的 AA 患者可能从延长 CsA 治疗中获益。鉴于 CsA 的副作用,停药时机值得进一步探讨。

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