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

Junbin Huang; Lifen Huang; Su Liu; Shaofen Lin; Yucai Cheng; Xiaoyun Jiang; Hongman Xue; Chikong Li; Chun Chen

doi:10.2147/IJGM.S313898

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

Int J Gen Med. 2021 Jul 2:14:3133-3144. doi: 10.2147/IJGM.S313898. eCollection 2021.

Authors

Junbin Huang^#¹, Lifen Huang^#¹, Su Liu², Shaofen Lin², Yucai Cheng¹, Xiaoyun Jiang³, Hongman Xue¹, Chikong Li⁴, Chun Chen¹

Affiliations

¹ Division of Hematology/Oncology, Department of Pediatrics, The Seventh Affiliated Hospital, Sun Yat-sen University, Shenzhen, 518107, People's Republic of China.
² Department of Pediatrics, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou, 510120, People's Republic of China.
³ Department of Pediatrics, The First Affiliated Hospital, Sun Yat-sen University, Guangzhou, 518000, People's Republic of China.
⁴ Division of Haematology/Oncology/BMT, Department of Paediatrics, The Chinese University of Hong Kong, Hong Kong, 999077, People's Republic of China.

^# Contributed equally.

Abstract

Objective: 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: aplastic anemia; immunosuppressive therapy; pediatric; predicting factor.

Grants and funding

This research was funded by Sanming Project of Medicine in Shenzhen (SZSM202011004), the National Natural Science Foundation of China (8157010694), Science, Technology and Innovation Commission of Shenzhen Municipality (JCYJ20180307150419435), Clinical Founding of The seventh affiliated hospital, Sun Yat-sen University(ZSQYLCKYJJ202024).