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

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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.

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Authors and Affiliations

  1. Department of Pediatrics, School of Medicine, Nihon University, 30-1 Ohyaguchi-Kamicho, Itabashi-ku, 173-8610, Tokyo, Japan

    Hiroshi Yagasaki & Hiroyuki Shichino

  2. Division of Blood Transfusion, Toho University Omori Hospital, Tokyo, Japan

    Akira Ohara

  3. Department of Pediatrics, Sapporo Hokuyu Hospital, Sapporo, Japan

    Ryoji Kobayashi

  4. Department of Cell Transplantation and Regenerative Medicine, Tokai University School of Medicine, Isehara, Japan

    Hiromasa Yabe

  5. Department of Perinatal and Pediatric Medicine, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan

    Shouichi Ohga

  6. Department of Pediatrics, Hiroshima Red Cross Hospital, Hiroshima, Japan

    Kazuko Hamamoto

  7. Department of Pediatrics, Hyogo College of Medicine, Hyogo, Japan

    Yoshitoshi Ohtsuka

  8. Department of Pediatrics, School of Medicine, Keio University, Tokyo, Japan

    Hiroyuki Shimada

  9. Department of Hematology/Oncology, Osaka Medical Center and Research Institute for Maternal and Child Health, Izumi City, Osaka, Japan

    Masami Inoue

  10. Department of Pediatrics, Graduate School of Medicine, Nagoya University, Nagoya, Japan

    Hideki Muramatsu, Yoshiyuki Takahashi & Seiji Kojima

Authors
  1. Hiroshi Yagasaki
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  2. Hiroyuki Shichino
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  3. Akira Ohara
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  4. Ryoji Kobayashi
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  5. Hiromasa Yabe
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  6. Shouichi Ohga
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  7. Kazuko Hamamoto
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  8. Yoshitoshi Ohtsuka
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  9. Hiroyuki Shimada
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  10. Masami Inoue
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  11. Hideki Muramatsu
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  12. Yoshiyuki Takahashi
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  13. Seiji Kojima
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Correspondence to Hiroshi Yagasaki.

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Yagasaki, H., Shichino, H., Ohara, A. et al. Immunosuppressive therapy with horse anti-thymocyte globulin and cyclosporine as treatment for fulminant aplastic anemia in children. Ann Hematol 93, 747–752 (2014). https://doi.org/10.1007/s00277-013-1984-x

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  • DOI: https://doi.org/10.1007/s00277-013-1984-x

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