Objective To explore the efficacy and safety of eltrombopag in the treatment of immune thrombocytopenia secondary to connective tissue diseases.Methods Totally 23 patients with immune associated thrombocytopenia secondary to connective tissue diseases who were treated with eltrombopag in the Department of Rheumatology,the First Affiliated Hospital of Soochow University,from January 2020 to December 2022 were selected as the treatment group.Additionally,34 patients who did not receive eltrombopag during the same period were collected as the control group.Statistical analysis was conducted using SPSS 26.0 software.The t test or Wilcoxon rank-sum test were used to compare differences between groups of categorical data.The chi-square test or Fisher's exact test was used to compare the differences between groups of quantitative data.Results The highest overall response rate of the 23 patients was 82.6％(19/23),which occurred at the 12th week after treatment.The blood platelet count was significantly increased from baseline at the first week[(47±29)×109/L,t=-5.25,P＜0.001],also at the 2nd week[(111±87)×109/L,t=-5.31,P＜0.001],the 4th week[(150±104)×109/L,t=-6.23,P＜0.001],the 12th week[(153±92)×109/L,t=-6.64,P＜0.001],the 24th week[(134±83)×109/L,t=-7.11,P＜0.001],and the difference was statistically significant.Compared with the conventional treatment group,patients in the eltrombopag group had a higher proportion of patients previously being treated with IVIG[60.9％(14/23)vs.20.6％(7/34),x2=9.57,P=0.002],a lower proportion of patients currently using IVIG[21.7％(5/23)vs.61.8％(21/34),x2=8.86,P=0.003],and a lower maximum daily dose of glucocorticoid use[80(50,120)mg/d vs.80(55,115)mg/d,Z=-2.02,P=0.042],the differences were statistically significant.After being followed up for 24 weeks,no patients experienced any serious adverse reactions related to eltrombopag.Conclusion Eltrombopag is effective and safe in the treatment of immune thrombocytopenia secondary to connective tissue diseases.It can be used as one of the choices for patients who failed to respond to conventional treatment or patients who could not tolerate high-dose glucocorticoids.
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