Combined administration of thrombopoietin receptor agonists and cyclosporin A for the treatment of transfusion-dependent non-severe aplastic anemia
CHEN Xiao-yu
YANG Hui
ZHANG Ting
GONG Yue-min
ZHANG Ya-wen
JIA Jin-song
SUN Jing-nan
HE Guang-sheng
Abstract：Objective To evaluate the safety and efficacy of thrombopoietin receptor agonists(TPO-RAs)in combination with Cyclosporin A(CsA)for the treatment of transfusion-dependent non-severe aplastic anemia(TD-NSAA).Methods A prospective analysis was conducted on 62 TD-NSAA patients who were registered at Chinese Eastern Collaboration Group of Anemia and administered herombopag(23 patients),eltrombopag(22 patients),or avatrombopag(17 patients)in combination with CsA from June 2018 to January 2024,and compared with a historical CsA monotherapy cohort(71 patients)to evaluate the efficacy and safety of TPO-RAs.Results In the TPO-RAs+CsA cohort and the CsA monotherapy cohort,the overall response rates at 3,6 and 12 months were 51.6％vs.18.3％,76.7％vs.28.6％,and 82.7％vs.52.3％(all P＜0.001),respectively.The complete response rates were 3.2％vs.1.4％(P=0.598),7.4％vs.1.4％(P=0.052),and 20.1％vs.7.0％(P=0.017),respectively.The 2-year cumulative severe aplastic anemia(SAA)progression rates were 5.7％and 36.4％,respectively(P=0.009).The 2-year cumulative event-free survival(EFS)rates were 52.1％and 40.4％,respectively(P=0.006).High absolute reticulocyte count(ARC)and the use of TPO-RAs were negatively correlated with the progression of SAA(P=0.041,P=0.008).There was no significant difference in response at 3,6,and 12 months after treatment among the three TPO-RAs subgroups(P＞0.05).There were two relapses in the herombopag group and two in the eltrombopag group,respectively.One death occurred from severe infection in the herombopag group and one in the avatrombopag group.Conclusion Herombopag,eltrombopag,or avatrombopag,in combination with CsA for the treatment of TD-NSAA,are more efficacious than CsA alone,and improve the prognosis of TD-NSAA,with no significant differences seen between the various types of TPO-RAs.
Keywords：transfusion-dependent non-severe aplastic anemiathrombopoietin receptor agonistsherombopageltrombopagavatrombopag
Publication Date：2025-01-31
Online Publishing Date：2025-08-15(First online date of this platform, not the publication date of the document)
Pages：6( 48-53 )