Clinical Outcome of Acquired Post-Immunosuppressive-Therapy Aplastic Anemia in Pediatric Patients: A 13-Year Experience in Two Southern China Tertiary Care Centers

Abstract

Junbin Huang,1,* Lifen Huang,1,* Su Liu,2 Shaofen Lin,2 Yucai Cheng,1 Xiaoyun Jiang,3 Hongman Xue,1 Chikong Li,4 Chun Chen1 1Division of Hematology/Oncology, Department of Pediatrics, The Seventh Affiliated Hospital, Sun Yat-sen University, Shenzhen, 518107, People’s Republic of China; 2Department of Pediatrics, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou, 510120, People’s Republic of China; 3Department of Pediatrics, The First Affiliated Hospital, Sun Yat-sen University, Guangzhou, 518000, People’s Republic of China; 4Division of Haematology/Oncology/BMT, Department of Paediatrics, The Chinese University of Hong Kong, Hong Kong, 999077, People’s Republic of China*These authors contributed equally to this workCorrespondence: Chun ChenDivision of Hematology/Oncology, Department of Pediatrics, The Seventh Affiliated Hospital, Sun Yat-sen University, No. 628 Zhenyuan Road, Guangming District, Shenzhen, 518107, People’s Republic of ChinaTel [phone]Email [email protected] LiDivision of Haematology/Oncology/BMT, Department of Paediatrics, The Chinese University of Hong Kong, 30-32 Ngan Shing Street, Shatin, New Territories, Hong Kong, 999077, People’s Republic of ChinaTel [phone]Email [email protected]: The aim of the present study is to evaluate the efficacy, complications, and contributing factors of immunosuppressive therapy (IST) response in children with acquired aplastic anemia (AA) and to explore optimal therapeutic methods for different clinical AA types.Methods: A total of 130 children diagnosed with acquired AA underwent IST in the Department of Pediatrics at Sun Yat-sen Memorial Hospital and the Department of Pediatrics at Seventh Affiliated Hospital, Sun Yat-sen University, between January 1, 2006, and July 15, 2020. The overall survival (OS), response rates, complications, and response predictors were analyzed. The response rates were compared according to clinical AA type.Results: All 130 children with AA were followed up with for a median of 50.6 months. Among the patients, 25 had non-severe AA (NSAA), 64 had severe AA (SAA), and 41 had very severe AA (VSAA). All patients initially received IST. In 13 patients, the IST failed; these patients received an allo-hematopoietic stem cell transplant as a salvage regimen. The OS rate was 90.3% ± 2.8%, and the response rates at 3, 6, 9, and 12 months were 34.19%, 39.32%, 49.57%, and 66.67%, respectively. The prolonged follow-up period might have led to higher response rates, especially in patients with SAA and VSAA. A multivariate logistic regression analysis of prognostic factors was conducted; the results showed that high red blood cell (RBC) and platelet (PLT) counts were associated with a high overall response rate and that the RBC count at diagnosis is a major contributing factor.Conclusion: With the use of rabbit anti-thymocyte globulin, proper cyclosporine management, and a prolonged IST follow-up period, a higher number of patients with acquired AA than normal achieved response. Proportionally, the number of patients who achieved remission within 12 months was higher in the SAA group (38.18%→ 63.64%) and VSAA group (28.95%→ 65.79%) than in the NSAA group (58.33%→ 75%). Higher RBC and PLT counts at diagnosis can predict a favorable outcome.Keywords: pediatric, aplastic anemia, immunosuppressive therapy, predicting factor

Keywords

• pediatric；aplastic anemia；immunosuppressive therapy；predicting factor