Development and validation of a predictive model to guide the use of plerixafor in pediatric population

Area of Science:

• Hematology
• Pharmacology

Background:

• Plerixafor, a CXCR4 receptor antagonist, mobilizes hematopoietic stem cells (HSCs) into peripheral blood.
• Accurate prediction of HSC harvest volume is crucial for successful transplantation.

Purpose of the Study:

• To develop and validate a predictive model for CD34+ cell harvest volume in pediatric patients.
• To assess the relationship between peripheral blood CD34+ cell counts and apheresis collection yield.

Main Methods:

• Data from 45 pediatric patients in the MOZAIC study were analyzed.
• Patients received granulocyte colony-stimulating factor (G-CSF) alone or with plerixafor.
• A linear regression model was used to correlate pre-apheresis peripheral blood CD34+ counts with apheresis collection (AP-CD34+).

Main Results:

• A strong, predictive linear relationship was found between pre-apheresis peripheral blood CD34+ counts and AP-CD34+ cell collection.
• The model predicts approximately 13 CD34+ cells collected for every 100 circulating pre-apheresis.
• The algorithm quantifies minimal pre-apheresis CD34+ counts for achieving target AP-CD34+ cell yields (2x10^6 or 5x10^6 cells/kg) with 90% assurance.

Conclusions:

• The developed predictive model accurately estimates CD34+ cell harvest volume in pediatric patients.
• This model can guide the strategic use of plerixafor in patients at risk of mobilization failure.
• The findings support optimizing HSC mobilization and collection protocols.