Severe congenital neutropenia and pegfilgrastim

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Novel treatment for severe congenital neutropenia with pegfilgrastim.

Severe congenital neutropenia (SCN) of infancy is a rare disorder with onset very early in life. Until the introduction of bone marrow transplantation as a curative strategy, and more recently with the introduction of recombinant granulocyte colony-stimulating factor (G-CSF), this was often a lethal disorder. Although G-CSF has prolonged survival, patients continue to die of infectious complica...

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Severe congenital neutropenia: case report

Introduction Neutropenia is defined in the literature as absolute neutrophil counts in peripheral blood of less than 1500 cells/mm3 in more than one year old and less than 2000cells/mm3 in children in the first year old of life. Neutropenia is classified as mild, moderate or severe, and may be congenital or acquired, persistent or not. Kostmann syndrome is a severe neutropenia, the incidence va...

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Granulopoiesis in severe congenital neutropenia.

The pathogenesis of the granulopoietic failure in three children with severe congenital neutropenia was studied. Mature neutrophils were absent from both peripheral blood and bone marrow. Assay of bone marrow granulocyte colony-forming cells (CFU-C) in a methylcellulose tissue culture system using colony-stimulating activity (CSA) from peripheral blood leukocytes demonstrated normal or increase...

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Pegfilgrastim for chemotherapy-induced neutropenia.

Many chemotherapy regimens cause myelosuppression, which can result in febrile neutropenia and potentially lead to serious infections. The risk of neutropenia and its complications can be reduced with filgrastim, a granulocyte-colony-stimulating factor. Filgrastim is safe and effective, but it is cleared rapidly from the body (predominantly through the kidneys) and requires daily administration...

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Severe congenital neutropenia: new lane for ELANE.

In this issue of Blood, Tidwell et al1 demonstrate that mutations in the start codon (protein synthesis is initiated at the codon ATG) of neutrophil elastase (ELANE) result in the production of N-terminally truncated elastase, which mislocates to the nucleus and results in severe congenital neutropenia (SCN).

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ژورنال

عنوان ژورنال: European Journal of Haematology

سال: 2009

ISSN: 0902-4441,1600-0609

DOI: 10.1111/j.1600-0609.2008.01145.x