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Implications of Dose and Response to Long-Term Granulocyte Colony Stimulating Factor (G-CSF) in a Patient with Severe Congenital Neutropenia

Description

Rosenberg et al evaluated patients with severe congenital neutropenia who received long-term therapy with granulocyte colony stimulating factor (G-CSF). A patient who responds poorly to higher doses is at greater risk for a poor outcome. The authors are from the National Cancer Institute (NCI) and multiple institutions in the United States, Canada, Aurstralia and Europe.


 

Parameters:

(1) dose of G-CSF relative to median dose

(2) absolute neutrophil count at 6 to 18 months (relative response to G-CSF)

Parameter

Finding

Points

dose relative to median

< 8 µg/kg/d

0

 

>= 8 µg/kg/d

1

absolute neutrophil count

>= 2,188 per µL

0

 

< 2,188 per µL

1

 

total score =

= SUM(points for the 2 parameters)

 

Interpretation:

• minimum score: 0

• maximum score: 2

• The higher the score the greater the risk of myelodysplasia with or without AML and the risk of dying from sepsis.

• A patient with both risk factors may benefit from an early hematopoietic stem cell transplant.

 


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