Efficacy of immunosuppressive therapy combined with recombinant human thrombopoietin on severe aplastic anemia in children / 中华实用儿科临床杂志

ABSTRACT

Objective To evaluate the impact of recombinant human thrombopoietin(rhTPO)in response of immunosuppressive therapy(IST)in children with severe aplastic anemia(SAA). Methods A retrospective analysis was made. The clinical data included 70 children with acquired aplastic anemia (AA)treatment with antithymocyte globulin(ATG)combined with Cyclosporine at Beijing Children's Hospital,Capital Medical University between January 2009 and December 2016. Thirty - six cases received standard IST(IST group),and 34 cases received standard IST combined with rhTPO (IST + rhTPO group ). The SPSS 19. 0 statistical package was used for data analysis. Results Three months after IST,the hematologic response rate in the IST + rhTPO group was much higher than that in the IST group [88. 2％(30 / 34 cases)vs. 33. 3％ (12 / 36 cases),and there was significant difference between 2 groups (χ2 = 21. 961,P = 0. 000). The hematologic response rate at 6 months,12 months and the endpoint of follow - up in the IST + rhTPO group were 76. 7％(23 / 30 cases),78. 9％(15 / 19 cases),70. 6％(24 / 34 cases),and which in the IST group were 63. 9％(23 / 36 cases),75. 1％(27 / 36 cases),75. 0％(27 / 36 cases),which were not significant difference between the 2 groups(all P > 0. 05). The median time of partial remission (PR)was 1. 5 months,and the median time of complete remission (CR)was 6 months in IST + rhTPO group. The median time of PR was 4 months,and the median time of CR was 14 months in the IST group. By the endpoint of follow - ups,the median time of platelet transfusion in-dependence(33 d)and red cell transfusion independence(39 d)in the rhTPO group were all shorter than those in the IST group(78. 0 d and 93. 5 d,respectively),and there were a significant differences between the 2 groups(all P <0. 05). By 3 months after IST,the level of platelet,hemoglobin and absolute reticulocyte counts(ARC)in the IST +rhTPO group were much higher than those in the IST group(48 × 109 / L vs.14 ×109 / L,104. 5 g/ L vs 66. 5 g/ L,60.34 × 109 / L vs. 50. 92 × 109 / L,respectively),and there were significant differences between the 2 groups (all P < 0. 05). There was no differences of those at 6 months,12 months after IST between the 2 groups(all P > 0. 05). The 3 - year overall survival(OS)rate was 100. 0％(34 / 34 cases)in the IST + rhTPO group,87. 5％(32 / 36 cases)in the IST group,and there was no significant difference between the 2 groups (P > 0. 05). Conclusions Three months of rhTPO treatment after IST could improve early hematologic response rate in children with newly diagnosed acquired SAA. It may speed up recovery of platelet and hemoglobin levels and had no significant impacts on long - term hematologic response rate,platelet or hemoglobin recovery and OS rate.