Li Ruixin, Zhou Jiongtao, Liu Zhengyuan, Chen Xi, Long Qiqiang, Yang Yan, Lin Shengyun, Jia Jinsong, He Guangsheng, Li JianYong
Department of Hematology, The First Affiliated Hospital of Nanjing Medical University, Jiangsu Province Hospital, Collaborative Innovation Center for Cancer Personalized Medicine, Nanjing, China.
Department of Hematology, The Second Hospital of Nanjing, Nanjing, China.
Front Immunol. 2022 May 26;13:884312. doi: 10.3389/fimmu.2022.884312. eCollection 2022.
Addition of eltrombopag (E-PAG) to intensive immunosuppressive therapy (IST) contributes to restoring hematopoiesis in patients with severe aplastic anemia (SAA). Used at relatively low doses in the East Asian population, the efficacies of E-PAG and the predictors for efficacy are not clear. We conducted a retrospective, multicenter study to analyze the efficacy and the possible predicting factors at 6 months in 58 adult SAA patients with rabbit ATG-based IST and E-PAG. The response rate and complete response rate at 6 months were 76% and 21%, respectively. The baseline reticulocyte percentage [area under a curve (AUC)=0.798, 95% confidence interval (CI) 0.640-0.956, =0.006], absolute reticulocyte count (ARC) (AUC =0.808, 95%CI 0.647-0.970, =0.004), red cell distribution width - coefficient of variation (RDW-CV) (AUC=0.722, 95%CI 0.494-0.950, =0.040), and absolute lymphocyte count (ALC) (AUC=0.706, 95%CI 0.522-0.890, =0.057) were highly predictive of response at 6 months. The tipping values of reticulocyte percentage, ARC, RDW-CV, and ALC were 0.45%, 7.36×10/L, 11.75%, and 1.06×10/L, respectively. The sensitivity and specificity of reticulocyte percentages were 81.6% and 66.7%; ARC were 86.8% and 66.7%, RDW-CV were 94.7% and 55.6%; ALC were 55.3% and 88.9%. At a median follow-up of 15.5 months, the 2-year cumulative overall survival was 92%. The baseline reticulocyte percentage, ARC, RDW-CV, and ALC were potential factors in predicting a favorable effect of rabbit-ATG based IST plus E-PAG in SAA patients of East Asia (ChiCTR2100045895).
http://www.chictr.org.cn/edit.aspx?pid=125480&htm=4, identifier ChiCTR2100045895.
在强化免疫抑制治疗（IST）中添加艾曲泊帕（E-PAG）有助于恢复重型再生障碍性贫血（SAA）患者的造血功能。在东亚人群中使用相对较低剂量时，E-PAG的疗效及疗效预测因素尚不清楚。我们进行了一项回顾性多中心研究，分析58例接受兔抗胸腺细胞球蛋白（ATG）为基础的IST及E-PAG治疗的成年SAA患者6个月时的疗效及可能的预测因素。6个月时的缓解率和完全缓解率分别为76%和21%。基线网织红细胞百分比[曲线下面积（AUC）=0.798，95%置信区间（CI）0.640 - 0.956，P = 0.006]、绝对网织红细胞计数（ARC）（AUC = 0.808，95%CI 0.647 - 0.970，P = 0.004）、红细胞分布宽度-变异系数（RDW-CV）（AUC = 0.722，95%CI 0.494 - 0.950，P = 0.040）和绝对淋巴细胞计数（ALC）（AUC = 0.706，95%CI 0.522 - 0.890，P = 0.057）对6个月时的缓解具有高度预测性。网织红细胞百分比、ARC、RDW-CV和ALC的临界点分别为0.45%、7.36×10⁹/L、11.75%和1.06×10⁹/L。网织红细胞百分比的敏感性和特异性分别为81.6%和66.7%；ARC分别为86.8%和66.7%，RDW-CV分别为94.7%和55.6%；ALC分别为55.3%和88.9%。中位随访15.5个月时，2年累积总生存率为92%。基线网织红细胞百分比、ARC、RDW-CV和ALC是预测东亚SAA患者兔ATG为基础的IST加E-PAG疗效良好的潜在因素（ChiCTR2100045895）。
http://www.chictr.org.cn/edit.aspx?pid=125480&htm=4，标识符ChiCTR2100045895。
