Immunosuppressive therapy for patients with Down syndrome and idiopathic aplastic anemia.
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.
Department of Pediatrics, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Showa-ku, Nagoya, 4668550, Japan.
Kyogo
K
Suzuki
Department of Pediatrics, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Showa-ku, Nagoya, 4668550, Japan.
Hideki
H
Muramatsu
Department of Pediatrics, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Showa-ku, Nagoya, 4668550, Japan.
Yusuke
Y
Okuno
Department of Pediatrics, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Showa-ku, Nagoya, 4668550, Japan.
Atsushi
A
Narita
Department of Pediatrics, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Showa-ku, Nagoya, 4668550, Japan.
Asahito
A
Hama
Department of Pediatrics, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Showa-ku, Nagoya, 4668550, Japan.
Yoshiyuki
Y
Takahashi
Department of Pediatrics, Japanese Red Cross Asahikawa Hospital, Asahikawa, Japan.
Makoto
M
Yoshida
Division of Hematology and Oncology, Shizuoka Children's Hospital, Shizuoka, Japan.
Yasuo
Y
Horikoshi
Division of Hematology and Oncology, Shizuoka Children's Hospital, Shizuoka, Japan.
Ken-Ichiro
K
Watanabe
Department of Pediatrics, Fujita Health University School of Medicine, Toyoake, Japan.
Kazuko
K
Kudo
Department of Pediatrics, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Showa-ku, Nagoya, 4668550, Japan. kojimas@med.nagoya-u.ac.jp.
Seiji
S
Kojima
D000741:Anemia, Aplastic / Q000209:etiology / Q000628:therapy*; D000961:Antilymphocyte Serum / Q000627:therapeutic use; D016026:Bone Marrow Transplantation; D002648:Child; D016572:Cyclosporine / Q000627:therapeutic use; D004314:Down Syndrome / Q000150:complications / Q000628:therapy*; D006801:Humans; D007165:Immunosuppression Therapy / Q000379:methods; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D016896:Treatment Outcome
D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine
Aplastic anemia; Down syndrome; Immunosuppressive therapy