Non-myeloablative transplantation for severe congenital neutropenia

Pediatr Blood Cancer. 2008 Apr;50(4):920-1. doi: 10.1002/pbc.21295.

Abstract

Severe Congenital Neutropenia is a rare condition characterized by a very low neutrophil count, which pre-disposes the affected child to recurrent bacterial infections. Treatment with granulocyte colony stimulating factor (G-CSF) has dramatically improved the prognosis of these children; but in patients who have become G-CSF refractory, hematopoeitic stem cell transplant is still the only effective curative treatment. We describe a patient who was unresponsive to escalating doses of G-CSF and underwent a successful reduced intensity conditioning, matched unrelated donor allograft resulting in cure.

Publication types

  • Case Reports

MeSH terms

  • Child, Preschool
  • Cyclosporine / therapeutic use
  • Graft vs Host Disease / drug therapy
  • Granulocyte Colony-Stimulating Factor / therapeutic use
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Infant
  • Infant, Newborn
  • Male
  • Neutropenia / congenital*
  • Neutropenia / therapy*
  • Transplantation Conditioning* / methods

Substances

  • Granulocyte Colony-Stimulating Factor
  • Cyclosporine