AUTHOR=Li Ruixin , Zhou Jiongtao , Liu Zhengyuan , Chen Xi , Long Qiqiang , Yang Yan , Lin Shengyun , Jia Jinsong , He Guangsheng , Li JianYong 

TITLE=Predicting Response of Severe Aplastic Anemia to Rabbit-Antithymocyte Immunoglobulin Based Immunosuppressive Therapy Combined With Eltrombopag

JOURNAL=Frontiers in Immunology

VOLUME=13

YEAR=2022

URL=https://www.frontiersin.org/journals/immunology/articles/10.3389/fimmu.2022.884312

DOI=10.3389/fimmu.2022.884312

ISSN=1664-3224

ABSTRACT=<p>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, <italic>P</italic>=0.006], absolute reticulocyte count (ARC) (AUC =0.808, 95%CI 0.647-0.970, <italic>P</italic>=0.004), red cell distribution width – coefficient of variation (RDW-CV) (AUC=0.722, 95%CI 0.494-0.950, <italic>P</italic>=0.040), and absolute lymphocyte count (ALC) (AUC=0.706, 95%CI 0.522-0.890, <italic>P</italic>=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<sup>9</sup>/L, 11.75%, and 1.06×10<sup>9</sup>/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).</p><sec><title>Clinical Trial Registration</title><p><uri xlink:href="http://www.chictr.org.cn/edit.aspx?pid=125480&amp;htm=4" xmlns:xlink="http://www.w3.org/1999/xlink">http://www.chictr.org.cn/edit.aspx?pid=125480&amp;htm=4</uri>, identifier ChiCTR2100045895.</p></sec>