Kostmann Disease Medication

Updated: Aug 24, 2017
  • Author: Peter N Huynh, MD; Chief Editor: Harumi Jyonouchi, MD  more...
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Medication Summary

History and physical examination is important in assessing the severity of acute infections and determining treatment. Fevers higher than 39°C and low monocyte count (< 100/μL) are associated with more serious infections. Oral antibiotic therapy can be used for superficial or ear/nose/throat infection in moderate neutropenia with close monitoring if inflammatory markers are low (C-reactive protein, < 15 mg/L).

Patients with severe congenital neutropenia and sepsis require hospitalization. Blood, urine, and infectious-site cultures, as well as chest radiography, should be performed as clinically indicated. Aggressive intravenous parenteral antibiosis with a combination of a third-generation cephalosporin and an aminoglycoside should be considered. If fevers persist beyond 48 hours, the addition of antifungal agents is recommended.

In patients with severe infections that are worrisome, G-CSF should be started at the known dose that patient responds to, or standard dose of 5 μg/kg/d. There is an induction phase with G-CSF to evaluate the response of individual, with an increase in absolute neutrophil count (ANC) (>1500/μL) and clinical improvement after 10-15 days. The initial daily dose is 5 μg/kg subcutaneously. If there is no response after 15 days, the daily dose is increased by 5 μg/kg. If the response is rapid or excessive (ANC >5000/μL), the dose is halved.


Colony-stimulating factors

Class Summary

These agents are used to stimulate neutrophil production and act as hematopoietic growth factors that stimulate the development of granulocytes. They are used to treat or prevent neutropenia. Glycosylated G-CSF is not FDA approved in the United States but is available in England.

Filgrastim (G-CSF, Neupogen, tbo-filgrastim, Granix, filgrastim-sndz, Zarxio)

Filgrastim is a recombinant methionyl human granulocyte colony-stimulating factor (G-CSF) (r-metHuG-CSF) consisting of a 175-amino acid protein with a molecular weight of 18,800 d. It is produced by Escherichia coli bacteria into which the human G-CSF gene is inserted. This protein has an amino acid sequence identical to the natural sequence predicted from human DNA sequence analysis, except for the addition of an N-terminal methionine necessary for expression in E coli. Because it is produced in E coli, the product is nonglycosylated and thus differs from G-CSF isolated from human cells.

Pegfilgrastim (Neulasta)

Recombinant pegylated-conjugated human G-CSF. Acts on hematopoietic cells to stimulate production, maturation, and activation of neutrophilsl. Increases migration and cytotoxicity of neutrophils.