title: Allogeneic stem cell transplantation in a patient with dyskeratosis congenita after conditioning with low-dose cyclophosphamide and anti-thymocyte globulin.
abstract: 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.
authors.affiliation: Section of stem cell transplant, Department of Pediatric Hematology-Oncology, KFSHRC, Riyadh, Saudi Arabia. mouhab@kfshrc.edu.sa
authors.forename: M
authors.initials: M
authors.lastname: Ayas
authors.forename: A
authors.initials: A
authors.lastname: Al-Musa
authors.forename: A
authors.initials: A
authors.lastname: Al-Jefri
authors.forename: A
authors.initials: A
authors.lastname: Al-Seraihi
authors.forename: M
authors.initials: M
authors.lastname: Al-Mahr
authors.forename: S
authors.initials: S
authors.lastname: Rifai
authors.forename: H
authors.initials: H
authors.lastname: El-Solh
mesh_terms: D000961:Antilymphocyte Serum / Q000008:administration & dosage; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage; D019871:Dyskeratosis Congenita / Q000628:therapy*; D006086:Graft vs Host Disease / Q000276:immunology / Q000517:prevention & control; D006680:HLA Antigens / Q000276:immunology*; D006650:Histocompatibility Testing; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D033581:Stem Cell Transplantation / Q000379:methods*; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous
chemical_list: D000961:Antilymphocyte Serum; D006680:HLA Antigens; D007166:Immunosuppressive Agents; D003520:Cyclophosphamide