Impaired Bone Metabolism in Glycogen Storage Disease Type 1 Is Associated with Poor Metabolic Control in Type 1a and with Granulocyte Colony-Stimulating Factor Therapy in Type 1b

2014 ◽  
Vol 81 (1) ◽  
pp. 55-62 ◽  
Author(s):  
D. Melis ◽  
R. Pivonello ◽  
M. Cozzolino ◽  
R. Della Casa ◽  
F. Balivo ◽  
...  
Keyword(s):  
Bone Metabolism ◽  
Storage Disease ◽  
Glycogen Storage Disease Type ◽  
Glycogen Storage ◽  
Disease Type ◽  
Granulocyte Colony Stimulating Factor ◽  
Colony Stimulating Factor ◽  
Factor Therapy ◽  
Stimulating Factor

Effect of Granulocyte-Colony Stimulating Factor in Glycogen Storage Disease Type IB

1993 ◽  
Vol 21 (5) ◽  
pp. 276-279 ◽  
Author(s):  
G V Zuccotti ◽  
R Longhi ◽  
P Flumine ◽  
A Stangalint ◽  
E Riva
Keyword(s):  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage Disease Type ◽  
Glycogen Storage ◽  
Polymorphonuclear Leucocyte ◽  
Disease Type ◽  
Polymorphonuclear Leucocytes ◽  
Granulocyte Colony Stimulating Factor ◽  
Colony Stimulating Factor ◽  
Stimulating Factor

Five children with glycogen storage disease type Ib (glycogenosis Ib), a metabolic defect associated with neutropenia and impairment of neutrophil function, were treated with granulocyte colony stimulating factor, a haemopoietic growth factor that induces a significant increase in polymorphonuclear leucocyte count in vivo. Recurrent bacterial or fungal infections were recorded in the three patients who had very low polymorphonuclear leucocyte counts. Experience at this centre indicates that the absolute number of polymorphonuclear leucocytes is more important than the alteration of their metabolic function in determining the susceptibility of the patients to infection. Granulocyte colony stimulating factor was effective at increasing polymorphonuclear leucocyte numbers (and restored function to some extent) in patients with glycogenosis Ib. This drug might be beneficial in cases of severe infection, in glycogenosis Ib patients with neutropenia.

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