Yang Y, Yang W R, Wu Z J, Zhao X, Zhang L, Jing L P, Zhou K, Li Y, Peng G X, Li Y, Li J P, Song L, Ye L, Fan H H, Zhang F K
Institute of Hematology and Blood Disease Hospital, CAMS & PUMC, Tianjin 300020, China.
Zhonghua Xue Ye Xue Za Zhi. 2016 Dec 14;37(12):1038-1043. doi: 10.3760/cma.j.issn.0253-2727.2016.12.006.
To explore the characteristics of delayed hematologic response in very/severe aplastic anemia (V/SAA) patients who were treated with immunosuppressive treatment (IST) as first-line approach, and investigate the rationality of early salvage treatment in refractory patients. The data of V/SAA patients front-line treated with IST were retrospectively analyzed. Delayed response was defined as acquiring hematologic response between 6 and 12 months after 1 course of IST. The clinical as well as hematologic characteristics of the delayed responded patients were investigated. Of the 533 patients, 45 (8.44%, 45/533) were delayed hematologic responders, which accounted for 29.03% (45/155) of the whole non-responders at 6 months. The quality of response in delayed responders analyzed at 12 months (χ=62.616, <0.001) and at the end of follow-up (χ=6.299, =0.043) was significantly worse than that of robust response group. There were more VSAA patients in delayed response group compared with robust response group (57.8% 38.3%, =0.013), and all the baseline absolute reticulocyte (ARC) count, ARC proportion and absolute neutrophil count (ANC) were much lower than that in delayed response group. Multivariate analysis about the above 2 groups showed that the baseline ARC count <10×10/L significanty reduced the chance of hematologic response within 6 months [=3.641(95% 1.718-7.719) , =0.001], and not any factor was found to predict delayed hematologic response in non-responders at 6 months. The 5-year overall survival of 76.50% (95% 71.6%-81.4%) and event free survival of 29.10%(95% 25.2%-33.0% ) in non-responders at 6 months, both were worse than 97.6% (95% 96.6%-98.6% ) and 84.0% (95% 81.1%-86.9% ) ( <0.001) of robust response group. The incidence of delayed hematologic response in V/SAA patients by IST is low. The quality of delayed response is not satisfactory and there is no effective means to predict the delayed response. It is reasonable to carry out salvage treatment as early as possible.
为探讨以免疫抑制治疗（IST）作为一线治疗方法的极重型/重型再生障碍性贫血（V/SAA）患者延迟血液学反应的特征，并研究难治性患者早期挽救治疗的合理性。对接受IST一线治疗的V/SAA患者的数据进行回顾性分析。延迟反应定义为在1个疗程IST后6至12个月获得血液学反应。研究延迟反应患者的临床和血液学特征。在533例患者中，45例（8.44%，45/533）为延迟血液学反应者，占6个月时全部未缓解患者的29.03%（45/155）。在12个月（χ=62.616，P<0.001）和随访结束时（χ=6.299，P=0.043）分析的延迟反应者的缓解质量明显差于快速缓解组。与快速缓解组相比，延迟反应组中VSAA患者更多（57.8%对38.3%，P=0.013），且所有基线绝对网织红细胞（ARC）计数、ARC比例和绝对中性粒细胞计数（ANC）均远低于延迟反应组。对上述两组的多因素分析表明，基线ARC计数<10×10⁹/L显著降低了6个月内血液学缓解的机会[比值比=3.641（95%可信区间1.718 - 7.719），P=0.001]，且未发现任何因素可预测6个月时未缓解患者的延迟血液学反应。6个月时未缓解患者的5年总生存率为76.50%（95%可信区间71.6% - 81.4%），无事件生存率为29.10%（95%可信区间25.2% - 33.0%），均低于快速缓解组的97.6%（95%可信区间96.6% - 98.6%）和84.0%（95%可信区间81.1% - 86.9%）（P<0.001）。IST治疗的V/SAA患者延迟血液学反应的发生率较低。延迟反应的质量不尽人意，且没有有效的方法来预测延迟反应。尽早进行挽救治疗是合理的。
