Michaël Philippe

TL;DR: Cidofovir is an effective therapy for BKV-HC but requires very precise renal function management to avoid toxicity, and needs to be investigated in randomized controlled trials.

TL;DR: The authors' novel MMopt algorithm accurately calculates busulfan intravenous dosage requirements to achieve target plasma exposures in children up to 18 years of age and 110 kg using only 2 blood samples per adjustment compared with 6–9 samples for standard noncompartmental dose calculations.

TL;DR: Therapeutic drug monitoring (TDM)-guided dosing to reach the conventional area under the concentration–time curve (AUC) target range of 900–1500 μmol min/L is associated with better outcomes and busulfan TDM combined with model-based dose adjustment was associated with an increased probability of AUC target attainment.

TL;DR: It is suggested that busulfan-associated VOD is in part a concentration-dependent reaction and may have important implications for busulfans dosing and therapeutic drug monitoring practice in HSCT children.

TL;DR: Significant busulfan intra-individual variability may occur in children who receive a HSCT and is hardly predictable, so performing TDM repeatedly over therapy appears to be the best way to accurately estimateBusulfan exposure and perform precision dosing.