| ObjectiveTo compare the short-term therapeutic effect of different regimens combining recombinant human thrombopoietin(rhTPO)with immunosuppressive therapy(IST)applied in adult severe aplastic anemia(SAA).MethodsA retrospective analysis performed on 61 adult SAA patients who received IST combined with rhTPO regimen in the Hematology Hospital of Chinese Academy of Medical Sciences from January to July 2015.Among them,18 patients received rhTPO once a day(continuous group),and 43 patients received rhTPO once every other day(inter-day group).The short-term efficacy was evaluated according to bone marrow hyperplasia and peripheral blood routine level at 3 and 6 months after IST treatment.The transfusion of blood products within 8 weeks after treatment was dynamically observed,and the hematopoietic recovery effect of rhTPO on red blood cells and platelets was analyzed respectively.The differences in length of hospital stay and medical expenditure in different dose groups were compared,and the adverse reactions after rhTPO application were recorded.All data were analyzed using SPSS 18.0 software package.Quantitative data are represented by median and range,and classification data are represented by quantity and relative frequency(%).The rank sum test of two independent samples was used to compare the age,course of disease,blood routine before treatment,hospitalization expenses and hospitalization time of the two groups.Pearson chi-square test was used to compare gender,VSAA/SAA,ATG/ALG,reaction rate and blood transfusion rate of the two groups;paired sample rank sum test was used to compare the elevated levels of HGB and PLT at 12 weeks after IST.P<0.05 indicated that the difference was statistically significant,and bilateral tests were taken.Results1.There was no significant difference in basic clinical features between the two groups before IST.At 3 and 6 months after IST,there was no significant difference in the total hematological response rate between the continuous group and the daytime group(3 months:50.0%vs 51.2%,P=0.934).6 months:77.8%vs 69.8%,P=0.525).The good hematological response rate(CR+ GPR)in the continuous group at 3 months after IST was significantly higher than that in the daytime group(38.9%vs 9.3%,P=0.011),and the difference was not statistically significant at 6 months(50.0%vs 34.9%,P=0.27).2.At 12 weeks after IST,HGB and PLT increased 15.5(3?3)g/L and 48.5(20? 66)×109/L in the continuous group and 6(1-37)g/L and 14(7?105)x109/L in the daytime group,respectively.There was no significant difference in HGB between the two groups(P=0.701),and the change of PLT was more obvious in the continuous group(P=0.002).3.The number of red blood cell transfusion detached from the two groups at 4 and 8 weeks after rhTPO was basically the same(P=0.736 and P=0.519,respectively).At 8 weeks,the number of patients with continuous platelet transfusion was significantly higher than that in daytime group[16(88.9%)vs 21(48.8%),P=0.003].For the amount of red blood cell transfusion,there was a statistically significant difference between the continuous group and the daytime group at the eighth week[0.24(0-2.00)U vs 1.51(07.00)U,P=0.049].4.At 6 weeks after rhTPO,platelet transfusion in the continuous group was less than that in the daytime group[0.06(0-1.00)U vs 1.07(0-3.00)U,P=0.047).At 8 weeks after rhTPO,red blood cell transfusion decreased by 0.24(0-2.00)U vs 1.51(0-7.00)U,P=0.049.5.There was no significant difference in medical expenses and hospitalization time between the two groups.6.The adverse reactions of the two groups of patients were mild.ConclusionsIt's more effective to promote hematopoietic recovery and reduce platelet transfusion dependence when rhTPO was daily used other than used every other day.Continuous application of rhTPO daily does not increase the incidence of adverse events.ObjectiveTo evaluate the therapeutic efficacy and safety of immunosuppressive therapy(IST)combined with recombinant human thrombopoietin(rhTPO)for severe aplastic anemia(SAA)in pediatric patients.MethodA retrospective case-control study conducted and the clinical data of 45 pediatric patients with de novo SAA admitted to the Anemia Diagnosis and Treatment Center of Chinese Academy of Medical Sciences&Blood Disease Hospital during the period from December 2010 to December 2016 were analyzed,including 26 males and 19 females,with a median age of 11(10-13)years.15 patients received IST combined with rhTPO regimen(rhTPO group).rhTPO was administered subcutaneously at 300 U/kg/d for 28 days.30 matched baseline historical cases(control group),using standard IST program.The curative effects of the two groups at 3,6 and 12 months were compared.The changes of blood product infusion volume within 3 months were dynamically recorded,and the peripheral blood lymphocyte subsets were regularly detected.Normal distribution data are represented by mean(range),and t test is used for comparison between groups;polarization distribution data were expressed as medians(P25,P75).Mann-Whitney U test was used for comparison between groups.Gender,disease severity and IST program were tested by ?2 test;the efficacy was compared by?2 test and Ridit analysis.P<0.05 indicated that the difference was statistically significant.Two groups of data were compared by nonparametric rank sum test.All data were analyzed using SPSS 18.0 software package.Result1,The cases of rhTPO group and control group with good hematological response(CR plus GPR)at 3,6 and 12 months were 6(40%),7(46.7%),13(86.7%)and 3(10.0%),7(23.3%),14(46.7%),respectively(P=0.048,P=0.270,P=0.010).ridit analysis showed that the OR of rhTPO group was higher than that of the control group at 6 and 12 months(P=0.035 and P=0.006,respectively).2.3 months after treatment,the platelet transfusion volume in rhTPO group at the 4th-6th week was lower than that in the control group(1.4U vs 2.9U,P=0.002).The amount of red blood cell transfusion was less than that in the control group from 8 to 10 weeks(0.8U vs 2.6U,P=0.026).3.There was no significant difference in the proportion of CD3+CD8+between the two groups at 3 and 6 months before and after treatment(P=0.153 and P=0.159,respectively).At 12 months,rhTPO combined with IST group was lower than IST group(P=0.038).4.No serious adverse reactions occurred in the rhTPO group.ConclusionApplication of rhTPO can improve the short-term therapeutic efficacy of IST for pediatric SAA,alleviate transfusion dependence,and has a good safety profile. |
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