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Updated: Sep 9, 2025
Busulfan as a Myelosuppressive Agent for Generating Stable High-level Bone Marrow Chimerism in Mice
Published on: April 1, 2015
Once-Daily Versus Four-Times-Daily Intravenous Busulfan with Therapeutic Drug Monitoring as Conditioning for
Safaa Bazbaz1, Irina Zaidman2, Ehud Even-Or2
1Department of Clinical Pharmacy, School of Pharmacy, Faculty of Medicine, Hebrew University of Jerusalem, P.O.Box 12272, Jerusalem 9112102, Israel.
Insights
Once-daily busulfan (BU1) and four-times-daily busulfan (BU4) dosing regimens show similar results in pediatric hematopoietic stem cell transplantation (HSCT). Both regimens achieved comparable event-free survival and adverse event rates in children undergoing HSCT.
Area of Science:
- Hematology
- Pediatric Oncology
- Transplantation Medicine
Background:
- Busulfan is a critical component of myeloablative conditioning for pediatric hematopoietic stem cell transplantation (HSCT).
- Dosing strategies for busulfan, including once-daily (BU1) versus multiple daily doses (BU4), are being evaluated for optimal outcomes.
Purpose of the Study:
- To compare the clinical outcomes of once-daily (BU1) versus four-times-daily (BU4) busulfan dosing in pediatric HSCT recipients.
- To assess event-free survival, engraftment times, and adverse events associated with different busulfan dosing regimens.
Main Methods:
- Retrospective analysis of 70 pediatric patients undergoing HSCT between June 2018 and October 2023.
- Patients were divided into two groups: 35 receiving BU1 and 35 receiving BU4.
- Primary endpoint was 100-day event-free survival (EFS); secondary endpoints included engraftment and adverse events like sinusoidal obstruction syndrome (SOS).
Main Results:
- No statistically significant difference in 100-day EFS between BU1 (88.6%) and BU4 (85.7%) groups (p=0.768).
- Comparable times to neutrophil and platelet engraftment were observed between the two regimens.
- Incidence of SOS (17.1% in both groups) and acute graft-versus-host disease (41.9% vs 40.0%) were similar, with no significant increase in liver enzymes.
Conclusions:
- Once-daily and four-times-daily busulfan regimens yield comparable clinical outcomes in pediatric HSCT.
- Both dosing strategies demonstrated similar efficacy and safety profiles regarding EFS, engraftment, and adverse events.
- Further prospective studies are recommended to confirm these findings in a larger cohort.
Abstract:
Background/Objectives: Busulfan is a key component of myeloablative conditioning regimens in hematopoietic stem cell transplantation (HSCT) for pediatric patients with acute myeloid leukemia, solid tumors, and certain non-malignant diseases. This study compares the clinical outcomes of once-daily (BU1) versus four-times-daily (BU4) busulfan dosing regimens in pediatric HSCT recipients. Methods: A retrospective analysis was conducted on 70 pediatric patients who underwent HSCT at Hadassah Medical Center between June 2018 and October 2023. Thirty-five patients received the BU4 regimen, and 35 received BU1. The primary endpoint was 100-day event-free survival (EFS). Results: There was no statistically significant difference in 100-day event-free survival between the BU1 group (88.6%) and the BU4 group (85.7%; p = 0.768). Similarly, no significant differences were found in time to neutrophil engraftment (p = 0.251) or platelet engraftment (p = 0.688). Sinusoidal obstruction syndrome (SOS) occurred in 17.1% of patients in each group. No significant differences were observed in the increase in liver enzyme levels (p = 1.0). The incidence of acute graft-versus-host disease was comparable between the groups (41.9% for BU1 vs. 40.0% for BU4; p = 0.878). Conclusions: Once-daily and four-times-daily busulfan regimens demonstrated comparable clinical outcomes in terms of efficacy and adverse events. Further prospective studies are needed to validate these findings.
