Simplifying Busulfan Conditioning in Children: Once Daily vs. Four-Times-Daily IV Dosing in Hematopoietic Cell Transplantation
5 affiliations
- 1Service d’Hématologie et d’Oncologie Pédiatrique, Hôpitaux Universitaires de Strasbourg, 67200 Strasbourg, France
- 2Service Pharmacie, Hôpitaux Universitaires de Strasbourg, 67200 Strasbourg, France
- 3Laboratoire de Pharmacologie et Toxicologie Neurocardiovasculaire, Unité de Recherche 7296, Faculté de Médecine de Maïeutique et des Sciences de la Santé, Centre de Recherche en Biomédecine de Strasbourg (CRBS), Université de Strasbourg, 67085 Strasbourg, France
- 4Laboratoire de Biochimie et Biologie Moléculaire, Secteur de Chromatographie et Spectrométrie de Masse, Hôpitaux Universitaires de Strasbourg, 67098 Strasbourg, France
- 5Laboratoire d’ImmunoRhumatologie Moléculaire, INSERM UMR_S 1109, LabEx Transplantex, Fédération de Médecine Translationnelle de Strasbourg, Université de Strasbourg, 67085 Strasbourg Cedex, France
Abstract
Background/Objectives: Busulfan is a key component of conditioning regimens for hematopoietic cell transplantation (HCT). Although once-daily (OD) intravenous administration is logistically advantageous over the conventional four-times-daily (QID) schedule, concerns remain regarding a potential increased risk of hepatic veno-occlusive disease (VOD or sinusoidal obstruction syndrome/SOS), particularly in children. Methods: We retrospectively reviewed all pediatric patients receiving busulfan-based conditioning with therapeutic drug monitoring (TDM) at a single center (January 2015–December 2024). The primary analysis compared VOD/SOS incidence within 100 days post-HCT, diagnosed by revised EBMT criteria, between the OD and allogeneic QID groups (QIDallo). Secondary analyses evaluated VOD/SOS severity, busulfan pharmacokinetic (PK) parameters, and clinical risk factors and compared the OD and total QID groups. Results: One hundred HCT procedures were included (36 OD, 64 QIDtot including 39 QIDallo). There was no statistically significant between-group difference (p = 0.777 for OD/QIDallo and p = 0.960 for OD/QIDtot), including after adjustment for age and disease. OD yielded higher Cmax than QIDallo and QID (p < 0.001), but PK parameters were not associated with VOD/SOS. Independent risk factors were age ≤5 years (p = 0.001) and thalassemia (p = 0.035). Conclusions: In this pediatric cohort, PK-guided OD busulfan was not associated with an increased VOD/SOS risk despite higher Cmax, supporting its use in children when TDM is implemented. Young age and thalassemia remained the principal determinants of VOD/SOS.
Related
- Population pharmacokinetics and model-informed precision dosing of busulfan in Chinese pediatric allogeneic hematopoietic stem cell transplantation: a prospective real-world cohort study
- Addition of thiotepa to total body irradiation-based conditioning in allogeneic hematopoietic stem cell transplantation for relapsed/refractory acute leukemia and lymphoma: a propensity score-matched retrospective study.
- Hematopoietic Cell Transplantation in Children with Sickle Cell Disease: A Single-Center Experience and Exploration of an Age Threshold for Post-Transplant Complications
- EFFICACY AND SAFETY OF HAPLOIDENTICAL HEMATOPOIETIC STEM CELL TRANSPLANTATION AS FIRST-LINE TREATMENT FOR SEVERE APLASTIC ANEMIA IN CHILDREN: A SYSTEMATIC REVIEW
- The role of allogeneic hematopoietic stem cell transplantation in adults with mature T‐cell lymphomas
BibTeX
@article{dzedzej2026simplifying,
title = {{Simplifying Busulfan Conditioning in Children: Once Daily vs. Four-Times-Daily IV Dosing in Hematopoietic Cell Transplantation}},
author = {Manon Dzedzej and Charlotte Nazon and Guillaume Becker and Amelia-Naomi Sabo and Véronique Kemmel and Catherine Paillard},
journal = {Journal of Clinical Medicine},
year = {2026},
month = nov,
doi = {10.3390/jcm15218754},
url = {https://www.mdpi.com/2077-0383/15/21/8754},
note = {CC BY 4.0},
}
RIS
TY - JOUR
TI - Simplifying Busulfan Conditioning in Children: Once Daily vs. Four-Times-Daily IV Dosing in Hematopoietic Cell Transplantation
AU - Manon Dzedzej
AU - Charlotte Nazon
AU - Guillaume Becker
AU - Amelia-Naomi Sabo
AU - Véronique Kemmel
AU - Catherine Paillard
PY - 2026
DA - 2026/11/09
JO - Journal of Clinical Medicine
DO - 10.3390/jcm15218754
UR - https://www.mdpi.com/2077-0383/15/21/8754
AB - Background/Objectives: Busulfan is a key component of conditioning regimens for hematopoietic cell transplantation (HCT). Although once-daily (OD) intravenous administration is logistically advantageous over the conventional four-times-daily (QID) schedule, concerns remain regarding a potential increased risk of hepatic veno-occlusive disease (VOD or sinusoidal obstruction syndrome/SOS), particularly in children. Methods: We retrospectively reviewed all pediatric patients receiving busulfan-based conditioning with therapeutic drug monitoring (TDM) at a single center (January 2015–December 2024). The primary analysis compared VOD/SOS incidence within 100 days post-HCT, diagnosed by revised EBMT criteria, between the OD and allogeneic QID groups (QIDallo). Secondary analyses evaluated VOD/SOS severity, busulfan pharmacokinetic (PK) parameters, and clinical risk factors and compared the OD and total QID groups. Results: One hundred HCT procedures were included (36 OD, 64 QIDtot including 39 QIDallo). There was no statistically significant between-group difference (p = 0.777 for OD/QIDallo and p = 0.960 for OD/QIDtot), including after adjustment for age and disease. OD yielded higher Cmax than QIDallo and QID (p < 0.001), but PK parameters were not associated with VOD/SOS. Independent risk factors were age ≤5 years (p = 0.001) and thalassemia (p = 0.035). Conclusions: In this pediatric cohort, PK-guided OD busulfan was not associated with an increased VOD/SOS risk despite higher Cmax, supporting its use in children when TDM is implemented. Young age and thalassemia remained the principal determinants of VOD/SOS.
SN - 2077-0383
ER -