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 Kojima

Research output: Contribution to journalArticlepeer-review

3 Citations (Scopus)


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.

Original languageEnglish
Pages (from-to)130-133
Number of pages4
JournalInternational Journal of Hematology
Issue number1
Publication statusPublished - 01-07-2016

All Science Journal Classification (ASJC) codes

  • Hematology


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