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Article

Novel mechanism of G-CSF refractoriness in patients with severe congenital neutropenia

8 Sept 2004

Abstract excerpt

Severe congenital neutropenia (SCN) is a rare disease diagnosed at or soon after birth, characterized by a myeloid maturation arrest in the bone marrow, ineffective neutrophil production, and recurrent infections. Most patients respond to treatment with granulocyte colony-stimulating factor (G-CSF), and the majority harbor mutations in the neutrophil elastase gene. In the subset of patients with SCN transforming...

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PMID: 15353486
DOI: 10.1182/blood-2004-07-2613

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