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

• DOI: 10.3760/cma.j.issn.0253-2727.2022.05.008
• Publication Date: 2022-05-14
• Authors: Xiangrong Hu; Xin Zhao; L Zhang; L P Jing; W R Yang; Y Li; L Ye; Kang Zhou; J P Li; Guangxin Peng; Huihui Fan; Y Li; Y Yang; Yuanyuan Xiong; F K Zhang
• Keywords: Univariate analysis; Aplastic anemia

Objective: To reassess the predictors for response at 6 months in patients with severe or very aplastic anemia (SAA/VSAA) who failed to respond immunosuppressive therapy (IST) 3 months. Methods: We retrospectively analyzed clinical data of 173 SAA/VSAA from 2017 2018 received IST and were classified as nonresponders Univariate multivariate logistic regression analysis used evaluate factors that could predict Results: showed 3-month hemoglobin (HGB) level (P=0.017) , platelet (PLT) (P=0.005) absolute reticulocyte count (ARC) (P<0.001) trough cyclosporine concentration (CsA-C0) (P=0.042) soluble transferrin receptor (sTfR) (P=0.003) improved value (ARC(△)) (sTfR(△)) related 6-month response. The results PLT (P=0.020) ARC(△) independent prognostic If was less than 6.9×10(9)/L, hematological rate low, regardless patient's count. Survival both 3-year overall survival (OS) [ (80.1±3.9) % vs (97.6±2.6) %, P=0.002] event-free (EFS) (31.4±4.5) (86.5±5.3) P<0.001] significantly lower those group. Conclusion: Residual hematopoietic indicators after are parameters. reflect whether bone marrow hematopoiesis is recovering degree recovery. A second treatment be performed sooner a low ARC(△).目的： 再评估影响免疫抑制治疗（IST）后3个月未获血液学反应的重型/极重型再生障碍性贫血（SAA/VSAA）患者6个月疗效的因素。 方法： 回顾性分析2017-2018年连续收治的173例初治行IST且治疗后3个月未获血液学反应的SAA/VSAA患者的临床资料，对IST后3个月时的临床特征和血液学参数进行再评估，通过单因素和多因素分析找出影响6个月疗效获得的相关指标。 结果： 单因素分析结果显示IST后3个月无效患者的HGB（P=0.017）、PLT（P=0.005）、网织红细胞绝对计数（ARC）（P<0.001）、环孢素A血药浓度谷值（CsA-C0）（P=0.042）、血清可溶性转铁蛋白受体（sTfR）（P=0.003）、网织红细胞绝对计数改善值（ARC(△)）（P<0.001）、血清可溶性转铁蛋白受体改善值（sTfR(△)）（P<0.001）与IST后6个月疗效有关。多因素分析结果显示PLT<10×10(9)/L（P=0.020）和ARC(△)<6.9×10(9)/L（P<0.001）是IST后3个月未获血液学反应患者6个月疗效的危险因素。IST后6个月未获血液学反应组3年总生存率[（80.1±3.9）%对（97.6±2.6）%，P=0.002]和无事件生存率[（31.4±4.5）%对（86.5±5.3）%，P<0.001]均明显低于获得血液学反应组。 结论： 对IST后3个月未获血液学反应的SAA/VSAA患者再评估以预测其6个月疗效非常重要；IST后3个月残存造血仍是影响预后的主要参数；ARC(△)可反应骨髓造血是否正在恢复及恢复的程度；IST后3个月无效患者若ARC(△)<6.9×10(9)/L，无论PLT为何值，IST后6个月的血液学反应率均较低。.