ArticleFrontiers in pharmacology2026
Impact of busulfan exposure stratification on transplant outcomes and immune reconstitution in children with severe aplastic anemia.
Article in Frontiers in pharmacology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Background: Severe aplastic anemia (SAA) is a life-threatening bone marrow failure disorder. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an established curative treatment for pediatric patients with SAA. Busulfan, a key component of conditioning regimens, has a narrow therapeutic index and marked interindividual pharmacokinetic variability. However, the impact of different first-dose busulfan exposure levels on long-term outcomes, particularly immune reconstitution, remains unclear in pediatric patients receiving a 2-day conditioning regimen. We evaluated the clinical significance of busulfan exposure stratification in pediatric patients with SAA undergoing allo-HSCT. Methods: This retrospective study included 40 pediatric patients with SAA who underwent allo-HSCT. Patients were stratified into a low-exposure group (first-dose area under the concentration-time curve [AUC Results: No significant differences were observed between the groups in neutrophil or platelet engraftment, incidence of graft-versus-host disease, hemorrhagic cystitis, engraftment syndrome, or short-term survival outcomes. All nine immune outcomes changed significantly over the first 12 months after transplantation, documenting pronounced temporal immune reconstitution (All adjusted Conclusion: First-dose busulfan AUC
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