Clinical Analysis Of 295 Adult Patients With Primary Immune Thrombocytopenia

Background and objective:Primary immune thrombocytopenia(ITP)is a common hemorrhagic disease of adults characterized by different degrees of bleeding,single thrombocytopenia and no clear secondary factors.At present,there are relatively little research on the epidemiology and treatment.The pathogenesis has not been fully clarified,which the underlying factors affecting bleeding and therapeutic responses have not been clear.The first-line treatment of ITP has still dominated by the traditional standard dose prednisone(PSL)and high dose dexamethasone(HDD)at home and abroad,and significant progresses in the effective rate and long-term remission rate have not been made.This paper investigated the bleeding symptoms of ITP and the relevant factors,evaluated the short-and-long-term efficacy and drug safety of different first-line regimens,and analyzed the possible prognostic factors affecting long-term efficacy.This study aimed to deepen the understanding of adult primary ITP,and to provide certain guidance for bleeding risk assessment,regimen selection and disease management.Methods:This study retrospectively researched the 295 cases of adults primary ITP patients treated in hematology department from January 2017 to January 2020 in the First Hospital of Jilin University.The relevant factors affecting bleeding and bleeding site were analyzed.The short-and-long-term efficacy were compared between HDD group and PSL group on newly diagnosed ITP,and between single corticosteroids and combination with recombinant human thrombopoietin(rhTPO)therapy.The prognostic factors affecting the long-term efficacy were discussed.And the adverse reactions of different regimens were compared.Results:1.Bleeding symptoms and relevant factors:Bleeding rate was 93.9%in 295 cases.The bleeding rate in the group of blood platelet count(BPC)<10×10~9/L was significant higher than the group of BPC≥10×10~9/L(95.6%vs 84.8%,P=0.005).The median bleeding score was 2(1～8).The linearity between BPC and bleeding score revealed negative correlation that bleeding score increased as BPC decreased(r=-0.525,P=0.009).The proportion of bleeding with secondary anemia was 25.6%,and secondary anemia rate of woman was significant higher than man at the age of<60(24.4%vs 11.9%,P=0.040).2.Bleeding site and relevant factors:The bleeding site with highest frequency was purpura(78.6%),while the bleeding site with lowest frequency was cerebral bleeding(2.4%).The linearity between age and the frequency of visceral hemorrhage revealed positive correlation that the incidence of visceral bleeding increased with age(r=0.783,P=0.037).3.Efficacy comparison between group HDD and group PSL on newly diagnosed ITP:Response rate had no significant difference between two group(P=0.093).The median time to response of group HDD was significantly shorter than group PSL(P=0.000),but there was no significant difference between complete response(CR),response(R)and no response(NR)(P=0.223).The overall response rate and CR rate had no significant difference respectively after 3 months,6 months and 12 months(P value<0.05),likewise in duration of response(DOR)(P=0.985).4.Efficacy comparison between single corticosteroids regimen and combination with rhTPO on newly diagnosed ITP:In group HDD,the time to response and the short-term and long-term efficacy had no significant difference between single and combination regimen(all of P value>0.05).However,in group PSL,the median time to response of combination was significantly shorter than single regimen(P=0.045),but no difference about the short-and-long-term efficacy(all of P value>0.05).In group HDD and PSL,the DOR between subgroup of single corticosteroids and combination had no significant difference(P=0.504,0.865).5.Analysis of prognostic factors affecting long-term efficacy of first-line therapy:Univariate analysis showed that initial efficacy was a prognostic factor for DOR(P=0.009).The factors that P<0.3 tested by univariate analysis were conducted by multivariate analysis,as a result,initial efficacy was the independent prognostic factors affecting DOR(HR:0.419;95%CI:0.226～0.778;P=0.006).The DOR of group initial CR was superior to the counterpart of group initial R(P=0.009).The prognostic factors of initial CR had not been found.6.Analysis of adverse reactions of first-line therapy:The most common adverse reaction was weight gain(28.7%).The group HDD had significantly lower frequency of adverse reactions than group PSL(13.3%vs 31.2%,P=0.001).Conclusions:1.The patients with blood platelet count<10×10~9/L had higher frequency of bleeding,and the severity of bleeding increased with the decrease of platelet count.Young and middle-aged female was more easily to secondary onset anemia.The bleeding symptoms of ITP performed mildly in general,which had lowest frequency of intracranial hemorrhage.The risk of visceral hemorrhage increased with age.2.In first-line therapy of newly diagnosed ITP,high dose dexamethasone regimen showed faster efficacy,but no advantages in short-and-long-term efficacy.Combined with rhTPO acquired faster efficacy in standard dose prednisone treatment,but no significant improvement in short-and-long-term efficacy was observed with either corticosteroids.3.In first-line therapy of newly diagnosed ITP,initial CR was the independent prognostic factors affecting longer duration of response.4.The adverse responses were generally mild.High dose dexamethasone regimen had relative low adverse reaction rate and relative high security.