Long‐term safety of treatment with recombinant human granulocyte colony‐stimulating factor (r‐metHuG‐CSF) in patients with severe congenital neutropenias Journal Article

  

Authors:	Bonilla, M. A.; Dale, D.; Zeidler, C.; Last, L.; Reiter, A.; Ruggeiro, M.; Davis, M.; Koci, B.; Hammond, W.; Gillio, A.; Welte, K.
Article Title:	Long‐term safety of treatment with recombinant human granulocyte colony‐stimulating factor (r‐metHuG‐CSF) in patients with severe congenital neutropenias
Abstract:	Congenital neutropenias include a heterogenous group of diseases characterized by a decrease in circulating neutrophils. In phase I/II/III studies in patients with severe congenital and cyclic neutropenia, treatment with recombinant human granulocyte colony-stimulating factor (r-metHuG-CSF) resulted in a rise in the absolute neutrophil counts (ANC) and a reduction in infections. We report the effects of long-term safety of subcutaneous r-metHuG-CSF administration in 54 patients (congenital n = 44, cyclic n = 10) treated for 4-6 years. A sustained ANC response was seen in 40/44 severe congenital neutropenia patients and 10/10 cyclic neutropenia patients. Two patients required an increase of >25% in dose to maintain a clinical response; one patient became refractory to therapy. A significant decrease in the incidence of severe infections and the need for intravenous antibiotics was noted. Significant adverse events noted which may or may not be related to therapy included: osteopenia (n = 15), splenomegaly (n = 12), hypersplenism (n = 1), vasculitis (n = 2), glomerulonephritis (n = 1), BM fibrosis (n = 2), MDS/leukaemia (n = 3), and transient inverted chromosome 5q with excess blasts (n = 1). R-metHuG-CSF has been well tolerated in the majority of patients and resulted in a longterm improvement in their clinical status.
Keywords:	leukemia; neutrophils; g-csf; cells; congenital neutropenia; cyclic neutropenia; kostmanns syndrome; infantile genetic agranulocytosis
Journal Title:	British Journal of Haematology
Volume:	88
Issue:	4
ISSN:	0007-1048
Publisher:	John Wiley & Sons
Date Published:	1994-12-01
Start Page:	723
End Page:	730
Language:	English
ACCESSION:	WOS:A1994PW37500009
DOI:	10.1111/j.1365-2141.1994.tb05110.x
PROVIDER:	wos
PUBMED:	7529539
Notes:	Article -- Source: Wos

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