Treatment of childhood aplastic anemia with antithymocyte globulin, management of side effects and long-term follow-up / 中华儿科杂志

ABSTRACT

<p><b>OBJECTIVE</b>To evaluate the efficacy of antithymocyte globulin (ATG) based immunosuppression therapy for childhood aplastic anemia, to reduce the adverse effects and to observe the long-term outcome.</p><p><b>METHOD</b>Thirty-five children with aplastic anemia (AA) were enrolled in this study. Six of the cases had very severe AA (VSAA), 11 had severe AA (SAA)-I, 8 had SAA-II and 10 had moderate AA (MAA). All these patients were treated with ATG plus Cyclosporin A (CSA). The following measures were taken during the ATG therapy infection of the patients had been controlled before ATG treatment. Comprehensive anti-allergic measures were implemented. Close attention was paid to the hemorrhage related with platelet reduction caused by ATG and severe infection of the patients.</p><p><b>RESULT</b>Shortly after the ATG usage, all the patients had a significant decrease of absolute peripheral lymphoblast count by more than 60 percent. With a mean follow-up time of 28 months, the total effective rate was 77.14% (27/35), significant response rate was 57.14%(20/35). There was no significant difference among VSAA, SAA and MAA groups in the response rate. Adverse reactions included the following (1) 48.6% (17/35) patients presented mild anaphylactoid reaction during the first day of ATG treatment; (2) 42.9%(15/35) cases presented serum sickness 5 - 11 days after the last dose of ATG with a mean duration of 3.6 days, all the patients were cured effectively with methylprednisolone; (3) 25.7% (9/35) patient's peripheral blood platelet count was reduced, might be caused by ATG, to below 10 × 10(9)/L, but no patient had severe hemorrhagic complication after platelet transfusion was performed; (4) 22.9%(8/35)of patients got infection within a month after ATG therapy, including 3 cases with clinical septicemia, all the 3 cases recovered after antibiotics treatment. There was no ATG treatment-related death in this series.</p><p><b>CONCLUSION</b>ATG is a very effective therapy for children with SAA and MAA. Comprehensive measures are needed to prevent and handle the side effects to avoid treatment-related death. Long-term supportive therapy and proper follow up contribute to the favourable outcomes of the patients.</p>