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

Pediatr Blood Cancer. 2007 Jul;49(1):103-4. doi: 10.1002/pbc.20696.

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.

Publication types

  • Case Reports

MeSH terms

  • Antilymphocyte Serum / administration & dosage
  • Child, Preschool
  • Cyclophosphamide / administration & dosage
  • Dyskeratosis Congenita / therapy*
  • Graft vs Host Disease / immunology
  • Graft vs Host Disease / prevention & control
  • HLA Antigens / immunology*
  • Histocompatibility Testing
  • Humans
  • Immunosuppressive Agents / therapeutic use*
  • Male
  • Stem Cell Transplantation / methods*
  • Transplantation Conditioning*
  • Transplantation, Homologous

Substances

  • Antilymphocyte Serum
  • HLA Antigens
  • Immunosuppressive Agents
  • Cyclophosphamide