Clinical features and oucomes of patients with pure red cell aplasia(PRCA)associated with large granular lymphocytic leukemia(LGLL)with STAT3 gene mutation
WANG Wei
LIU Xiao-qing
CHENG Xiao-yu
ZHANG Ting
GONG Yue-min
YE Bao-dong
WU Xue-mei
SUN Jing-nan
CHENG Xi
SONG Yu-hua
LONG Qi-qiang
Abstract：Objective To explore clinical features and oucomes of patients with pure red cell aplasia(PRCA)associated large granular lymphocytic leukemia(LGLL)with STAT3 gene mutation.Methods 81 patients with LGLL-related PRCA from January 2018 to July 2023(registered in CECGA)were analyzed.STAT3 gene mutation screening was performed in patients with LGLL-associated PRCA,and the clinical features,outcomes of immunosuppresive therapy were analyzed.Results In this study,21 patients were diagnosed with PRCA associated with LGLL with STAT3 gene mutation,15 patients(71％)had a exon21 gene mutation,4 patients(19％)had a exon 20 gene mutation,1 patient had exon 20 and 21 gene mutations,1 patient had a exon 13 gene mutation.Y640F was high frequency of mutations site(42.9％).In patients treated by cyclosporin(CsA),complete remission rate(CRR)and overall response rate(ORR)were 31.3％and 56.3％,respectively.Patients who received cyclophosphamide and prednison(CP)treatment have higher CRR,ORR(53.8％vs.31.3％,P=0.274,84.6％vs.56.3％,P=0.130),and lower recurrence rate(77.8％vs.18.2％,P=0.022).Conclusion Exon 21 of STAT3 gene is a high frequency of mutations and Y640F is the most common mutation site in LGLL-PRCA patients with STAT3 mutations.Compared to CsA,patients treated by CP regimen achieve better efficacy and lower recurrence rate.
Keywords：STAT3 gene mutationlarge granular lymphocytic leukemiapure red cell aplsiaCyclosporineCyclophosphamideefficacy
Publication Date：2024-04-01
Online Publishing Date：2025-08-15(First online date of this platform, not the publication date of the document)
Pages：5( 321-325 )