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Dataset Information

Population pharmacokinetic modelling of busulfan and the influence of body composition in paediatric Fanconi anaemia patients.


ABSTRACT:

Aims

Fanconi anaemia (FA) is a rare disorder characterized by progressive bone marrow failure that requires haematopoietic cell transplantation (HCT). Busulfan is used in conditioning regimens prior to HCT. Doses used in non-FA patients cause life-threatening toxicities in FA patients and data on busulfan pharmacokinetics (PK) in this population are limited. This study characterized busulfan PK in paediatric FA patients using population PK modelling and evaluated the effect of body composition on steady-state concentrations (Css ).

Methods

A total of 200 busulfan plasma concentrations in 29 FA patients from a recent study (Clinicaltrials.gov; NCT01082133) were available for population PK modelling. The effect of different body size-scaled doses and body compositio

SUBMITTER: van Hoogdalem MW 

PROVIDER: S-EPMC7163375 | biostudies-literature | 2020 May

REPOSITORIES: biostudies-literature