Immunosuppressive therapy for patients with Down syndrome and idiopathic aplastic anemia
- 320 Downloads
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.
KeywordsAplastic anemia Down syndrome Immunosuppressive therapy
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.