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International Journal of Hematology

, Volume 104, Issue 1, pp 130–133 | Cite as

Immunosuppressive therapy for patients with Down syndrome and idiopathic aplastic anemia

  • Kyogo Suzuki
  • Hideki Muramatsu
  • Yusuke Okuno
  • Atsushi Narita
  • Asahito Hama
  • Yoshiyuki Takahashi
  • Makoto Yoshida
  • Yasuo Horikoshi
  • Ken-ichiro Watanabe
  • Kazuko Kudo
  • Seiji KojimaEmail author
Case Report

Abstract

Idiopathic aplastic anemia (AA) is a rare hematological complication of Down syndrome (DS). The safety and efficacy of immunosuppressive therapy (IST) in individuals with DS remain unknown. We used a standard regimen of IST, comprising antithymocyte globulin and cyclosporine A, to treat three children with DS and idiopathic acquired AA. Two patients achieved a hematological (complete or partial) response and became transfusion independent at the final follow-up. The third patient failed to respond to IST and underwent bone marrow transplantation from a human leukocyte antigen (HLA)-mismatched unrelated donor. None of the patients experienced severe or unexpected adverse events during IST. Our experience suggests that IST is a safe and reasonable treatment, even in individuals with DS who suffer from AA and lack an HLA-matched sibling donor.

Keywords

Aplastic anemia Down syndrome Immunosuppressive therapy 

Notes

Acknowledgments

The authors would like to thank all of the patients and their families. The authors would also like to thank Ms. Yoshie Miura, Ms. Yuko Imanishi, and Ms. Hiroe Namizaki for their valuable assistance.

Compliance with ethical standards

Conflict of interest

S.K. was supported by a grant from Sanofi K.K. The other authors declare that they have no conflict of interest. A summary of relevant information will be published with the manuscript.

Supplementary material

12185_2016_1997_MOESM1_ESM.docx (76 kb)
Supplementary material 1 (DOCX 76 kb)

References

  1. 1.
    Bruwier A, Chantrain CF. Hematological disorders and leukemia in children with Down syndrome. Eur J Pediatr. 2012;171:1301–7.CrossRefPubMedGoogle Scholar
  2. 2.
    Erdogan G, Aksoy M, Dincol K. A case of idiopathic aplastic anaemia associated with trisomy-21 and partial endoreduplication. Acta Haematol. 1967;37:137–42.CrossRefPubMedGoogle Scholar
  3. 3.
    Furuya A, Ishida M, Hodohara K, Yoshii M, Okuno H, Horinouchi A, et al. Epstein-Barr virus-related post-transplant lymphoproliferative disorder occurring after bone marrow transplantation for aplastic anemia in Down’s syndrome. Int J Clin Exp Pathol. 2014;7:438–42.PubMedGoogle Scholar
  4. 4.
    Gathwala G, Dalal P, Dalal JS, Choudhry O. Transient aplastic anemia in Down’s syndrome—a rare association. Eur J Med Genet. 2011;54:341–2.CrossRefPubMedGoogle Scholar
  5. 5.
    Hanukoglu A, Meytes D, Fried A, Rosen N, Shacked N. Fatal aplastic anemia in a child with Down’s syndrome. Acta Paediatr Scand. 1987;76:539–43.CrossRefPubMedGoogle Scholar
  6. 6.
    McWilliams NB, Dunn NL. Aplastic anemia and Down’s syndrome. Pediatrics. 1982;69:501–2.PubMedGoogle Scholar
  7. 7.
    Pavithran K, Raji NL. Aplastic anemia in Down’s syndrome. Am J Hematol. 2003;73:213.CrossRefPubMedGoogle Scholar
  8. 8.
    Rubin CM, Mick R, Johnson FL. Bone marrow transplantation for the treatment of haematological disorders in Down’s syndrome: toxicity and outcome. Bone Marrow Transplant. 1996;18:533–40.PubMedGoogle Scholar
  9. 9.
    Weinblatt ME, Higgins G, Ortega JA. Aplastic anemia in Down’s syndrome. Pediatrics. 1981;67:896–7.PubMedGoogle Scholar
  10. 10.
    Hasle H, Clemmensen IH, Mikkelsen M. Risks of leukaemia and solid tumours in individuals with Down’s syndrome. Lancet. 2000;355:165–9.CrossRefPubMedGoogle Scholar
  11. 11.
    Jeong DC, Chung NG, Cho B, Zou Y, Ruan M, Takahashi Y, et al. Long-term outcome after immunosuppressive therapy with horse or rabbit antithymocyte globulin and cyclosporine for severe aplastic anemia in children. Haematologica. 2014;99:664–71.CrossRefPubMedPubMedCentralGoogle Scholar
  12. 12.
    Yoshida N, Yagasaki H, Hama A, Takahashi Y, Kosaka Y, Kobayashi R, et al. Predicting response to immunosuppressive therapy in childhood aplastic anemia. Haematologica. 2011;96:771–4.CrossRefPubMedPubMedCentralGoogle Scholar
  13. 13.
    Goto H, Kaneko T, Shioda Y, Kajiwara M, Sakashita K, Kitoh T, et al. Hematopoietic stem cell transplantation for patients with acute lymphoblastic leukemia and Down syndrome. Pediatr Blood Cancer. 2015;62:148–52.CrossRefPubMedGoogle Scholar
  14. 14.
    Weijerman ME, de Winter JP. Clinical practice. The care of children with Down syndrome. Eur J Pediatr. 2010;169:1445–52.CrossRefPubMedPubMedCentralGoogle Scholar
  15. 15.
    Garre ML, Relling MV, Kalwinsky D, Dodge R, Crom WR, Abromowitch M, et al. Pharmacokinetics and toxicity of methotrexate in children with Down syndrome and acute lymphocytic leukemia. J Pediatr. 1987;111:606–12.CrossRefPubMedGoogle Scholar
  16. 16.
    Taub JW, Ge Y. Down syndrome, drug metabolism and chromosome 21. Pediatr Blood Cancer. 2005;44:33–9.CrossRefPubMedGoogle Scholar

Copyright information

© The Japanese Society of Hematology 2016

Authors and Affiliations

  • Kyogo Suzuki
    • 1
  • Hideki Muramatsu
    • 1
  • Yusuke Okuno
    • 1
  • Atsushi Narita
    • 1
  • Asahito Hama
    • 1
  • Yoshiyuki Takahashi
    • 1
  • Makoto Yoshida
    • 2
  • Yasuo Horikoshi
    • 3
  • Ken-ichiro Watanabe
    • 3
  • Kazuko Kudo
    • 4
  • Seiji Kojima
    • 1
    Email author
  1. 1.Department of PediatricsNagoya University Graduate School of MedicineNagoyaJapan
  2. 2.Department of PediatricsJapanese Red Cross Asahikawa HospitalAsahikawaJapan
  3. 3.Division of Hematology and OncologyShizuoka Children’s HospitalShizuokaJapan
  4. 4.Department of PediatricsFujita Health University School of MedicineToyoakeJapan

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