Non-myeloablative transplantation for severe congenital neutropenia.
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.
Thachil
Jecko
J
Department of Paediatric Haematology and Oncology, Royal Liverpool Children's Hospital, Eaton Road, Liverpool, United Kingdom.
Caswell
Mark
M
Bolton-Maggs
Paula H B
PH
Pizer
Barry
B
Keenan
Russell
R
D002675:Child, Preschool; D016572:Cyclosporine / Q000627:therapeutic use; D006086:Graft vs Host Disease / Q000188:drug therapy; D016179:Granulocyte Colony-Stimulating Factor / Q000627:therapeutic use; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D009503:Neutropenia / Q000151:congenital* / Q000628:therapy*; D019172:Transplantation Conditioning / Q000379:methods
D016179:Granulocyte Colony-Stimulating Factor; D016572:Cyclosporine