The goal of this pilot study is to determine if scaling the first busulfan dose to Body Surface Area (BSA) in children with a BSA ≥0.5 m2 and using a BSA-banded dosing table for infants (BSA \<0.5 m2) increases the fraction of patients achieving a therapeutic drug exposure after the first dose.
Subject age: ≤21 years
Group A: BSA ≥0.5 m2
Group B: BSA <0.5 m2
Planned for once-daily busulfan-containing conditioning regimen pre-bone marrow transplant
None
Single group
Busulfan (BU)
DrugBusulfan is a cell cycle non-specific alkylating agent which is approved by the Food and Drug Administration (FDA) and is commercially available.
Number of participants who achieve a therapeutic drug exposure after the first dose of Busulfan
Dosing for patients in Group A (BSA \>=0.5m2) will be scaled to Body Surface Area, and patients in Group B (BSA \<0.5m2) will be dosed based on the infant dosing table for Day 1 dose. The value range for therapeutic drug exposure is 36,000 μM/min (Lower Bound) - 6,000 μM/min (Upper Bound)
Glutathione sample concentration over the 4-day course of busulfan administration.
Plasma glutathione samples will be obtained daily to determine glutathione concentration over the 4 days busulfan is administered. A 4-day total of gluathione sample concentration will be reported.
Number of participants with sinusoidal obstruction
Target adverse events (SOS, engraftment failure) will be tracked in all patients for the first 100 days post-transplant.
Number of participants with engraftment failure
Target adverse events (SOS, engraftment failure) will be tracked in all patients for the first 100 days post-transplant.
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Children's Hospital of Philadelphia
Lead sponsor
Alex's Lemonade Stand Foundation
Collaborator
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