Toggle Main Menu Toggle Search

Open Access padlockePrints

Stem cell transplantation in patients with severe congenital neutropenia without evidence of leukemic transformation

Lookup NU author(s): Dr Terence Flood, Professor Roderick Skinner


Full text for this publication is not currently held within this repository. Alternative links are provided below where available.


Severe congenital neutropenia (CN) (Kostmann syndrome) is a hematologic disorder characterized by a maturation arrest of myelopoiesis at the promyelocyte/myelocyte stage of development, This arrest results in severe neutropenia leading to absolute neutrophil counts (ANC) below 0.2 x 10(9)/L associated with severe bacterial infections from early infancy. Data on over 300 patients with CN collected by the Severe Chronic Neutropenia International Registry (SCNIR) beginning in 1994 indicate that more than 90% of these patients respond to recombinant human granulocyte colony stimulating factor (r-HuG-CSF) treatment with an ANC greater than 1.0 x 10(9)/L. For patients who are refractory to r-HUG-CSF treatment and continue to have severe and often life-threatening bacterial infections, hematopoietic stem cell transplantation is the only currently available treatment. We report on a total of 11 patients with CN reported to the SCNIR who underwent transplantation for reasons other than malignant transformation between 1976 and 1998. Of these patients, 8 were nonresponders or showed only partial response to r-HuG-CSF treatment with ongoing infections. Results from these patients suggest that transplantation of stem cells from an HLA-identical sibling is beneficial for patients refractory to r-HuG-CSF. (Blood. 2000;95:1195-1198) (C) 2000 by The American Society of Hematology.

Publication metadata

Author(s): Zeidler C, Welte K, Barak Y, Barriga F, Bolyard AA, Boxer L, Cornu G, Cowan MJ, Dale DC, Flood T, Freedman M, Gadner H, Mandel H, O'Reilly RJ, Ramenghi U, Reiter A, Skinner R, Vermylen C, Levine JE

Publication type: Article

Publication status: Published

Journal: Blood

Year: 2000

Volume: 95

Issue: 4

Pages: 1195-1198

ISSN (print): 0006-4971

ISSN (electronic): 1528-0020