Clinical Efficacy Of Cyclosporine A With Stanozolol In The Treatment Of 30 Children Patients With Chronic Aplastic Anemia

[Objective]To investigate the efficacy and safety of combination of cyclosporine A with stanozolol in children with chronic aplastic anemia(CAA).[Methods]Thirty children patients received combination of cyclosporine A (3mg/Kg/d) and stanozolol (0.14mg/Kg. d)treatment. Twenty-seven/thirteen children were treated and followed at least twelve months. The whole blood routine test, transfusion dependence and bone marrow smears were investigated and all the adverse effects were recorded to evaluate the treatment efficacy and tolerance.[Results] Twenty-seven cases of the thirty cases were finished the twelve-month treatment. Initial dose of cyclosporine A was 3mg/kg/d. After three months the dose were increased to 4mg/kg/d in five cases according to the level of cyclosporine A in serum. Eight cases (26.6%) were cured and acquired the treatment targets at the end of the 6th month. Ten cases (33.3%) were stable and got the treatment targets at the 2nd month. Six cases (20.0%) improved significantly and got treatment targets at the 2nd month. Six cases had no improvement. Five cases depended on red blood cell transfusions. Seven cases depended on platelet transfusions and two cases depended on both blood cell and platelet transfusions after the treatment. Bone marrow hyperplasia activity was improved in terms of increased erythroid, megakaryocyte and decreased lymphatic system in the eight cases. bone marrow index were normal in 6 cured cases with increased ratio of CFU-E(+EPO),BFU-E(+EPO),CFU-GM in bone marrow stem cells culture. bone marrow hyperplasia activity had no significant change in six invalid cases'. But lymphatic system decreased and erythroid increased in these six patients. The bone marrow stem cells were not improved compared to initial. Three patients were lost to follow in six months, seven months, eleven months during the treatment. Twteny-seven patients finished the over twelve months treatment. The treatment regime was adjusted because of liver toxicl in one case and low voice in another case. One case developed dysuria duo to clitoris increase. the symptoms improved after changing the treatment of stanozolol. gingival hyperplasia occurred and improved after low dose oral corticosteroids in six cases fur increase and low voice were developed to varying degrees in other patients during the treatment, which can be tolerated. Most of patients tolerated the adverse effects and did not discontinue the treatment.[Conclusions]The combination of cyclosporine A with stanozolol is a safe and effective in children with chronic aplastic anemia. It is needed further study with large samples.