Published in:
01-07-2016 | Case Report
Immunosuppressive therapy for patients with Down syndrome and idiopathic aplastic anemia
Authors:
Kyogo Suzuki, Hideki Muramatsu, Yusuke Okuno, Atsushi Narita, Asahito Hama, Yoshiyuki Takahashi, Makoto Yoshida, Yasuo Horikoshi, Ken-ichiro Watanabe, Kazuko Kudo, Seiji Kojima
Published in:
International Journal of Hematology
|
Issue 1/2016
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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.