Efficacy, safety and prognosis analysis of oral cyclosporine immunosuppressive regimen in children with acquired non-severe aplastic anemia: 13 years’ experience from a children’s hematology center of China
Authors: Fu Yang , Huang Zhongping , Wang Hongsheng , Zhu Xiaohua , Cao Ping , Yu Yi , Qian Xiaowen , Shen Chen , Gao Xuan , Zhai Xiaowen 
Published: February 2024

Background
Aplastic anemia (AA) is a serious hematopoietic system disease in children. Although there is no standard treatment scheme for acquired non-severe aplastic anemia (NSAA) at present, cyclosporine (CsA) has always been the most commonly used first-line treatment drug for children NSAA. The number of children with AA is large in China. This study aimed to explore the effect, safety and prognosis analysis of CsA on Chinese children with acquired NSAA.
Methods
The clinical data of children with acquired NSAA who were diagnosed in the affiliated pediatric hospital of Fudan University from December 2010 to June 2023 and whose initial treatment was mainly oral CsA treatment (excluding the use of anti-thymocytes/anti-lymphocyte globulin) were analyzed.
Results
A total of 40 NSAA patients were enrolled, including 24 males and 16 females, with a median age of 7.0 years (2.5–16.5 years). Among them, 5 cases (12.5%) were blood transfusion dependent, including 1 case of red blood cell (RBC) dependence, 1 case of platelet (Plt) dependence, and 3 cases of both RBC and Plt dependence. Complete remission (CR) was achieved in 9 patients (22.5%), partial remission (PR) in 21 patients (52.5%), and none remission (NR) in 10 patients (25.0%). In PR patients, 5 patients progressed during treatment, and received hematopoietic stem cell transplantation (HSCT), 4 patients underwent hematopoietic reconstitution, and 1 patient died after transplantation; Among 10 NR patients, 4 patients progressed to SAA, of which 2 patients were still treated with oral medicine, and the other 2 patients were treated with HSCT for disease progression 2 and 4 years after treatment (1 patient died of viral brainstem encephalitis combined with sepsis after transplantation); One NR patient died of severe agranulocytosis; The other 5 cases were relatively stable after CsA treatment. The RBC count in CR group and PR group was significantly higher than that in NR group (P < 0.01 and P < 0.05, respectively). Hemoglobin (Hb) in CR group and PR group was higher than that in NR group (P < 0.05). Plt count in CR group was higher than that in NR group (P < 0.05). The average dosage of CsA in NR group was significantly higher than that in CR group (P < 0.05). The peak plasma concentration of CsA in NR group was significantly higher than that in CR group (P < 0.05). The increase of white blood cell (WBC) in CR patients and PR patients after 60 days of CsA treatment was higher than that in NR patients (P < 0.01). The Plt count of CR patients was higher than that of CR and PR patients (P < 0.01). There was no significant difference in WBC, ANC, absolute reticulocyte count (ARC), cellular immune function and CsA blood grain concentration at diagnosis among CR, PR and NR patients, and there was no difference in the increased values of ANC, RBC, Hb and ARC at 60 days after treatment.
Conclusions
CsA is an effective drug for the treatment of acquired NSAA in children, with good safety under therapeutic drug monitoring (TDM). The efficacy is related to the levels of RBC, Hb and Plt at the time of diagnosis and the response of WBC and Plt at 60 days after treatment.