ArticleWorld journal of pediatrics : WJP2026
Safety of sirolimus in pediatric vascular anomalies: a BKMR-PopPK exposure-response framework for individualized risk and dosing.
Article in World journal of pediatrics : WJP, 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
backgroundVascular anomalies are rare, heterogeneous disorders that threaten the health of children. Sirolimus is an important drug therapy, but its high incidence of adverse drug reactions (ADRs) may cause treatment discontinuation and therefore poor disease control. Although prior studies suggest a strong link between sirolimus exposure and ADRs, the quantitative relationship remains unclear. This study aimed to systematically quantify the relationships between steady-state trough sirolimus concentrations (C
methodsWe retrospectively analyzed the data using Bayesian kernel machine regression (BKMR) to characterize associations of C
resultsA total of 257 patients were involved in this study (766-892 blood samples). BKMR results showed the quantitative exposure-response relationships between C
conclusionsThis is the first study to quantify the relationship between C
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