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[重新评估免疫抑制治疗三个月后无血液学反应的重度或极重度再生障碍性贫血患者的六个月预后]

[Reassessing the six months prognosis of patients with severe or very severe aplastic anemia without hematological responses at three months after immunosuppressive therapy].

作者信息

Hu X R, Zhao X, Zhang L, Jing L P, Yang W R, Li Y, Ye L, Zhou K, Li J P, Peng G X, Fan H H, Li Y, Yang Y, Xiong Y Z, Zhang F K

机构信息

State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China.

出版信息

Zhonghua Xue Ye Xue Za Zhi. 2022 May 14;43(5):393-399. doi: 10.3760/cma.j.issn.0253-2727.2022.05.008.

Abstract

To reassess the predictors for response at 6 months in patients with severe or very severe aplastic anemia (SAA/VSAA) who failed to respond to immunosuppressive therapy (IST) at 3 months. We retrospectively analyzed the clinical data of 173 patients with SAA/VSAA from 2017 to 2018 who received IST and were classified as nonresponders at 3 months. Univariate and multivariate logistic regression analysis were used to evaluate factors that could predict the response at 6 months. Univariate analysis showed that the 3-month hemoglobin (HGB) level (=0.017) , platelet (PLT) level (=0.005) , absolute reticulocyte count (ARC) (<0.001) , trough cyclosporine concentration (CsA-C0) (=0.042) , soluble transferrin receptor (sTfR) level (=0.003) , improved value of reticulocyte count (ARC(△)) (<0.001) , and improved value of soluble transferrin receptor (sTfR(△)) level (<0.001) were related to the 6-month response. The results of the multivariate analysis showed that the PLT level (=0.020) and ARC(△) (<0.001) were independent prognostic factors for response at 6 months. If the ARC(△) was less than 6.9×10(9)/L, the 6-month hematological response rate was low, regardless of the patient's PLT count. Survival analysis showed that both the 3-year overall survival (OS) [ (80.1±3.9) % (97.6±2.6) %, =0.002] and 3-year event-free survival (EFS) [ (31.4±4.5) % (86.5±5.3) %, <0.001] of the nonresponders at 6 months were significantly lower than those of the response group. Residual hematopoietic indicators at 3 months after IST are prognostic parameters. The improved value of the reticulocyte count could reflect whether the bone marrow hematopoiesis is recovering and the degree of recovery. A second treatment could be performed sooner for patients with a very low ARC(△).

摘要

重新评估在3个月时对免疫抑制治疗(IST)无反应的重度或极重度再生障碍性贫血(SAA/VSAA)患者6个月时反应的预测因素。我们回顾性分析了2017年至2018年173例接受IST且在3个月时被归类为无反应者的SAA/VSAA患者的临床资料。采用单因素和多因素逻辑回归分析来评估可预测6个月时反应的因素。单因素分析显示,3个月时的血红蛋白(HGB)水平(=0.017)、血小板(PLT)水平(=0.005)、绝对网织红细胞计数(ARC)(<0.001)、环孢素谷浓度(CsA-C0)(=0.042)、可溶性转铁蛋白受体(sTfR)水平(=0.003)、网织红细胞计数改善值(ARC(△))(<0.001)以及可溶性转铁蛋白受体改善值(sTfR(△))水平(<0.001)与6个月时的反应相关。多因素分析结果显示,PLT水平(=0.020)和ARC(△)(<0.001)是6个月时反应的独立预后因素。如果ARC(△)低于6.9×10⁹/L,无论患者的PLT计数如何,6个月时的血液学反应率都较低。生存分析显示,6个月时无反应者的3年总生存(OS)[(80.1±3.9)% 对 (97.6±2.6)%,=0.002]和3年无事件生存(EFS)[(31.4±4.5)% 对 (86.5±5.3)%,<0.001]均显著低于反应组。IST后3个月的残余造血指标是预后参数。网织红细胞计数的改善值可反映骨髓造血是否恢复及恢复程度。对于ARC(△)非常低的患者可更早进行二次治疗。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/072d/9250949/77bb70f38dd7/cjh-43-05-393-g001.jpg

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