Long-term outcomes of 95 children with moderate aplastic anemia treated with horse antithymocyte globulin and cyclosporine

Eri Nishikawa, Hiroshi Yagasaki, Asahito Hama, Hiromasa Yabe, Akira Ohara, Yoshiyuki Kosaka, Kazuko Kudo, Ryoji Kobayashi, Shouichi Ohga, Akira Morimoto, Ken Ichiro Watanabe, Nao Yoshida, Hideki Muramatsu, Yoshiyuki Takahashi, Seiji Kojima

Research output: Contribution to journalArticlepeer-review

9 Citations (Scopus)


Background: Currently, the standard management of moderate aplastic anemia (MAA) has not been well described, although the superiority of the combination of antithymocyte globulin (ATG) and cyclosporine (CyA) over CyA alone has been demonstrated in terms of hematological responses and failure-free survival (FFS). Procedure: We adopted this therapeutic strategy and treated 95 children with MAA who were enrolled in two consecutive prospective studies between October 1992 and August 2009. Results: For these patients, the 6-month response rate was 54.7% (complete response, 13.7%; partial response, 41.1%). There were no statistically significant differences in the overall response rates between the transfusion-dependent (48.8%, n = 41) and transfusion-independent groups (59.3%, n = 54; P = 0.4). Treatment failure was defined as the requirement of salvage treatment, and was observed in 52 patients. The 10-year FFS was 44.0% (95% confidence interval [CI], 32.9%–54.6%). Of the 22 patients who underwent a second immunosuppressive therapy (IST), 12 responded. Forty patients underwent hematopoietic stem cell transplantation as second- or third-line therapy and three died of complications. Consequently, the 10-year overall survival rate was 96.0% (95% CI, 88.0%–98.7%) with a median follow-up period of 103 months (range, 29–221 months). Conclusions: Although current guidelines recommend only observation for patients with transfusion-independent MAA, the results of our study justify early intervention with ATG and CyA in those patients. A prospective randomized trial is warranted to clarify the risks and benefits of early intervention with IST and observation alone until progression to severe AA in patients with MAA.

Original languageEnglish
Article numbere26305
JournalPediatric Blood and Cancer
Issue number5
Publication statusPublished - May 1 2017
Externally publishedYes

All Science Journal Classification (ASJC) codes

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology


Dive into the research topics of 'Long-term outcomes of 95 children with moderate aplastic anemia treated with horse antithymocyte globulin and cyclosporine'. Together they form a unique fingerprint.

Cite this