PubMed · 16533744
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Shinji Nakao, H Joachim Deeg, Takayuki Ishikawa, Judith Marsh, Alan List, Masao Tomonaga. 2005. Myelodysplastic syndrome.. https://doi.org/10.1532/ijh97.05139
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Bone marrow failure is the major cause of early mortality in patients with dyskeratosis congenita (DC); early trials with conventional conditioning regimens were associated with remarkable chronic morbidity and mortality, and the optimal conditioning regimen for these patients remains undetermined. We report a case of a child afflicted with DC who underwent related full HLA-matched stem cell transplant (SCT) using a regimen of low dose cyclophosphamide and antithymocyte globulin (ATG). The regimen was well tolerated and associated with no significant short-term toxicity.
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