Allogeneic stem cell transplantation in a patient with dyskeratosis congenita after conditioning with low-dose cyclophosphamide and anti-thymocyte globulin

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 (ATIG). The regimen was well tolerated and associated with no significant short-term toxicity.