ArticleBiology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation2013
Effect of weight and maturation on busulfan clearance in infants and small children undergoing hematopoietic cell transplantation.
Article in Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 43 papers, 2 of them syntheses that pooled it.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
43 citing papers in PubMed, 2 syntheses or guidelines pooled it, 78 citations in OpenAlex.
- Population Pharmacokinetic Model of Intravenous Busulfan in Hematopoietic Cell Transplantation: Systematic Review and Comparative Simulations.Clinical pharmacokinetics · 2023Pooled it
- Clinical pharmacology of cytotoxic drugs in neonates and infants: Providing evidence-based dosing guidance.European journal of cancer (Oxford, England : 1990) · 2022Pooled it
- Lentiviral Gene Therapy for Artemis-Deficient SCID.The New England journal of medicine · 2022Trial
- IV and oral fosfomycin pharmacokinetics in neonates with suspected clinical sepsis.The Journal of antimicrobial chemotherapy · 2021Trial
- Personalized pharmacokinetic targeting with busulfan in allogeneic hematopoietic stem cell transplantation in infants with acute lymphoblastic leukemia.International journal of hematology · 2019Trial
- Low Exposure Busulfan Conditioning to Achieve Sufficient Multilineage Chimerism in Patients with Severe Combined Immunodeficiency.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2019Trial
- Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.The New England journal of medicine · 2019Trial
- Population pharmacokinetics and pharmacodynamics of the artesunate-mefloquine fixed dose combination for the treatment of uncomplicated falciparum malaria in African children.Malaria journal · 2019Trial
- From Therapeutic Drug Monitoring to Model-Informed Precision Dosing: A Review of Busulfan Dosing Optimization in Pediatric Hematopoietic Stem Cell Transplantation.Pharmaceutics · 2026Review
- Updated EBMT/ESID inborn errors working party guidelines for haematopoietic stem cell transplantation for inborn errors of immunity and metabolism.Bone marrow transplantation · 2026Review
- Pharmacokinetics and safety of fosfomycin and flomoxef administered as part of neonatal sepsis treatment (NeoSep1 Part 1).Antimicrobial agents and chemotherapy · 2026Article
- New approach to busulfan dosing in infants and children based on a population pharmacokinetic analysis.Cancer chemotherapy and pharmacology · 2025Article
- Semi-mechanistic population pharmacokinetic model incorporating glutathione S-transferase activity for personalized busulfan dosing in pediatric allogeneic hematopoietic cell transplantation.Frontiers in pharmacology · 2025Article
- Article
- The Correlation Between Busulfan Exposure and Clinical Outcomes in Chinese Pediatric Patients: A Population Pharmacokinetic Study.Frontiers in pharmacology · 2022Article
- Donor Killer Immunoglobulin Receptor Gene Content and Ligand Matching and Outcomes of Pediatric Patients with Juvenile Myelomonocytic Leukemia Following Unrelated Donor Transplantation.Transplantation and cellular therapy · 2021Article
- Precision dosing of intravenous busulfan in pediatric hematopoietic stem cell transplantation: Results from a multicenter population pharmacokinetic study.CPT: pharmacometrics & systems pharmacology · 2021Article
- Evaluation of two software using Bayesian methods for monitoring exposure and dosing once-daily intravenous busulfan in paediatric patients receiving haematopoietic stem cell transplantation.Cancer chemotherapy and pharmacology · 2021Article
- Vitamin D levels and busulphan kinetics in patients undergoing hematopoietic stem cell transplantation, a multicenter study.Bone marrow transplantation · 2021Article
- SCID newborn screening: What we've learned.The Journal of allergy and clinical immunology · 2021Review
Corrections and comments
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Authors and funding
13 authors at 10 institutions in 5 countries.
Funding
Abstract
Little information is currently available regarding the pharmacokinetics (PK) of busulfan in infants and small children to help guide decisions for safe and efficacious drug therapy. The objective of this study was to develop an algorithm for individualized dosing of i.v. busulfan in infants and children weighing ≤12 kg, that would achieve targeted exposure with the first dose of busulfan. Population PK modeling was conducted using intensive time-concentration data collected through the routine therapeutic drug monitoring of busulfan in 149 patients from 8 centers. Busulfan PK was well described by a 1-compartment base model with linear elimination. The important clinical covariates affecting busulfan PK were actual body weight and age. Based on our model, the predicted clearance of busulfan increases approximately 1.7-fold between 6 weeks to 2 years of life. For infants age <5 months, the model-predicted doses (mg/kg) required to achieve a therapeutic concentration at steady state of 600-900 ng/mL (area under the curve range, 900-1350 μM·min) were much lower compared with standard busulfan doses of 1.1 mg/kg. These results could help guide clinicians and inform better dosing decisions for busulfan in young infants and small children undergoing hematopoietic cell transplantation.
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