Is antithymocyte globulin effective in the treatment of medium-sized reentry in children?

Aplastic anemia (AA; abbreviated as remyelination) is caused by damage to bone marrow stem cells and/or the hematopoietic microenvironment due to chemical, physical, and biological factors as well as unknown causes, resulting in centripetal atrophy of the red marrow, which is replaced by the fatty marrow, and a decrease in the number of peripheral whole blood cells. The detailed pathogenesis is still unclear, but the involvement of immune injury in pathogenesis has been recognized by most scholars. Immunosuppressive agents have been used clinically to treat reentry, including antithymocyte globulin, cyclosporine and cyclophosphamide (CTX) [1-5], especially ATG combined with CSA, which has been widely used in the treatment of severe reentry with good efficacy. In recent years, we have used it for the treatment of medium-sized recurrent cataract in children. In China, cataracts are generally classified as acute cataracts and chronic cataracts, and in recent years, we have refined the classification of cataracts into heavy cataracts, medium-sized cataracts, light cataracts, and temporary cataracts by synthesizing foreign standards, which is conducive to a more targeted choice of treatment options. Generally speaking, bone marrow transplantation or combined immunosuppressive therapy is used for severe cataracts, while observation and supportive therapy are used for mild and temporary cataracts. On the other hand, less research has been done on the treatment of medium-sized cataracts, especially in children, and supportive therapy and clinical observation are the mainstays of treatment, with immunosuppression or bone marrow transplantation only practiced in the case of severe cataracts. There are no controlled studies of early treatment versus non-treatment of medium-sized cataracts.Scott retrospectively analyzed 24 cases (median age 8 years) of children with medium-sized cataracts treated with supportive therapy only and followed up for 10-293 months (mean 66 months).16 cases (68%) developed severe cataracts requiring combined immunosuppressive and bone marrow transplantation; 3 cases (12.5%) resolved on their own; and 5 cases (20.8%) remained as medium-sized reocclusion. The time of transition to severe remodeling was variable, within 3 months of diagnosis of MAA in 5 cases, while in 2 cases it was one or two decades later. A study of 12 cases of MAA in children followed up from 1978 to 1991 from St. Jude Children’s Research Hospital reported that 3 cases did not require treatment, 4 were transfusion dependent and 5 developed SAA.The high incidence of conversion of MAA to SAA indirectly suggests that effective treatment regimen should be adopted early in children with medium sized remodeling.The data of our cases treated with immunosuppressive therapy showed that the conversion to severe remodeling occurred in 30.23% (13/43, 2 cases in the ATG group and 11 cases in the non-ATG group), which is lower than the non-treatment literature (68%). Studies on the treatment of non-severe in cataracts have been seen mainly in adults. Treatment of 16 patients (median age 38 years) with medium-sized cataracts with recombinant human anti-interleukin II receptor antibody (daclizumab) has been reported; 38% (6/16) of the patients responded to the treatment, with one relapsed patient still responding to a second daclizumab treatment. In the remaining cases, only one case progressed after two years of follow-up [11].Marsh [12] reported in a randomized, multicenter, prospective study of non-severe cataracts that 61 patients (median age 35 years) received CSA, and 54 patients (median age 29 years) were treated with ATG and CSA. In the CSA group, complete response was 23% and partial response was 23%; in the ATG plus CSA group, complete and partial response was 57% and 17%, respectively. And the mean hemoglobin level and platelet count of ATG plus CSA group were higher than that of CSA group. In this data, 18 cases of children with medium-sized cataracts were treated with ATG+CSA+androgen, and 25 cases were treated only with CSA+androgen, with a total effective rate of 52.38% in both groups. Both the total effective rate and the apparent rate were higher in the ATG group, the non-transfusion-dependent survival rate was higher than that in the non-ATG group, and the mortality rate of those who turned into severe remittent cataracts was significantly lower than that in the non-ATG group. We believe that it is best to use combination immunotherapy including ATG at an early stage for children with medium-sized cataracts.