Immunosuppressive therapy with horse anti-thymocyte globulin and cyclosporine as treatment for fulminant aplastic anemia in children

Hiroshi Yagasaki, Hiroyuki Shichino, Akira Ohara, Ryoji Kobayashi, Hiromasa Yabe, Shouichi Ohga, Kazuko Hamamoto, Yoshitoshi Ohtsuka, Hiroyuki Shimada, Masami Inoue, Hideki Muramatsu, Yoshiyuki Takahashi, Seiji Kojima

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4 Citations (Scopus)

Abstract

Patients with severe aplastic anemia (SAA) and an absolute neutrophil count (ANC) of 0 typically have fatal outcomes. We defined fulminant AA (FAA) as ANC=0 for at least 2 weeks prior to and after immunosuppressive therapy (IST). We analyzed the outcomes of 35 children with FAA among 288 children who enrolled in a prospective study for AA (AA-97 study). AA was classified as FAA (n=35), very SAA (vSAA; n=129), or SAA (n=124). All of the children received the IST with horse anti-thymocyte globulin (ATG) and cyclosporine (CsA). A significantly lower response rate at 6 months was seen in children with FAA when compared to those with vSAA or SAA (40.0, 63.6, and 63.7 %, respectively; p=0.027). Of 20 nonresponder patients in the FAA group, 11 were rescued by alternative donor transplantation, and 5 patients showed a late response after 6 months. Consequently, no significant difference was noted in overall survival when comparing the FAA, vSAA, and SAA groups (88.5, 95.8, and 96.8 %). These findings indicate that IST with ATG and CsA is justified as a first-line treatment for children with FAA who lack a human leukocyte antigen-matched sibling donor.

Original languageEnglish
Pages (from-to)747-752
Number of pages6
JournalAnnals of Hematology
Volume93
Issue number5
DOIs
Publication statusPublished - 2014 May

Keywords

  • Children
  • Cyclosporine
  • Fulminant aplastic anemia
  • Horse anti-thymocyte globulin
  • Immunosuppressive therapy

ASJC Scopus subject areas

  • Hematology

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    Yagasaki, H., Shichino, H., Ohara, A., Kobayashi, R., Yabe, H., Ohga, S., Hamamoto, K., Ohtsuka, Y., Shimada, H., Inoue, M., Muramatsu, H., Takahashi, Y., & Kojima, S. (2014). Immunosuppressive therapy with horse anti-thymocyte globulin and cyclosporine as treatment for fulminant aplastic anemia in children. Annals of Hematology, 93(5), 747-752. https://doi.org/10.1007/s00277-013-1984-x