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Surveillance of colony-stimulating factor use in US academic health centers
J M Yim1, K A Matuszewski, L C Vermeulen
1Clinical Practice Advancement Center, University Hospital Consortium, Oak Brook, IL 60521, USA.
Insights
Hematopoietic colony-stimulating factor (CSF) use in US academic centers showed significant costs for unproven indications. Further research is needed to validate promising uses and clarify dosing and safety for G-CSF and GM-CSF.
Area of Science:
- Hematology
- Pharmacoeconomics
- Health Services Research
Background:
- Hematopoietic colony-stimulating factors (CSFs), including G-CSF and GM-CSF, are critical in managing neutropenia.
- Their utilization in academic health centers warrants evaluation for appropriateness and cost-effectiveness.
Purpose of the Study:
- To characterize and evaluate the use of hematopoietic colony-stimulating factors (CSFs) in US academic health centers.
- To assess the cost implications associated with CSF therapy.
Main Methods:
- An observational study evaluated 565 patients receiving G-CSF or GM-CSF across 30 US academic health centers.
- Appropriateness of CSF use was assessed based on indication guidelines and FDA labeling, considering indication, type of CSF, and dosage.
Main Results:
- 71% of CSF use was appropriate by indication, but only 51% was appropriate by dosage.
- Substantial costs ($791,000) were associated with CSF therapy, with significant portions allocated to unproven or inappropriate uses.
- Adverse events were more common with GM-CSF (22%) than G-CSF (3.4%).
Conclusions:
- Significant financial resources are spent on CSF therapy lacking robust literature support.
- Further studies are essential to justify promising but unproven CSF indications and to clarify optimal dosing, monitoring, and safety profiles.
Objective:
To characterize and evaluate hematopoietic colony-stimulating factor (CSF) use, including cost implications, in US academic health centers.
Design:
An observational study of patients who received granulocyte colony-stimulating factor (G-CSF) or granulocyte-macrophage colony-stimulating factor (GM-CSF) from September 1 to October 15, 1993.
Setting:
Thirty academic health centers in the US.
Participants:
Five hundred sixty-five patients were evaluated.
Main Outcome Measures:
The appropriateness of CSF use was assessed, based on consensus-derived indication guidelines and the Food and Drug Administration-approved product labeling. Indication, type of CSF, and dosage were considered in determining the appropriateness of CSF therapy.
Results:
Based on indication evaluation criteria, 71% of CSF use was appropriate, 7% was inappropriate, and 22% was unproven, although the majority of unproven use was deemed promising by the expert panel. Based on dosage evaluation criteria, 51% of CSF use was appropriate, 27% was inappropriate, and 22% was for promising and other unproven indications. More than 90% of the patients studied received G-CSF. Approximately 3.4% of patients who received G-CSF had an adverse event, compared with 22% of those who received GM-CSF. Approximately $791,000 was spent on CSF therapy in the 565 patients: $401,000 (51%) on appropriate indications and doses, $160,000 (20%) on inappropriate doses for appropriate indications, $124,000 (16%) on promising indications, and $106,000 (13%) on unproven or inappropriate indications.
Conclusions:
Substantial costs are incurred currently for CSF therapy without adequate literature support. Further studies are warranted to justify promising but unproven uses of CSFs, as well as to clarify proper dosing, monitoring, and relative safety of CSFs.