Quantitative assessment of the exposure-efficacy relationship of glucocerebrosidase using Markovian elements in Gaucher patients treated with enzyme replacement therapy.

Elena Gras-ColomerVictor Mangas SanjuanMaría-Amparo Martínez-GómezMónica Climente-MartíMatilde Merino-Sanjuan

Published in: British journal of clinical pharmacology (2022)

A population pharmacokinetic model successfully characterized the leukocyte activity-time profiles of GCase following intravenous administration of ERT in GD1 patients together with an exposure-efficacy relationship in bone marrow using Markovian elements. The information obtained from this study could be of high clinical relevance in individualization of ERT in GD1 patients, as this could lead to anticipative decision-making regarding clinical response in bone and optimal dosing strategy.

Keyphrases

replacement therapy
bone marrow
end stage renal disease
ejection fraction
newly diagnosed
decision making
prognostic factors
mesenchymal stem cells
patient reported outcomes
body composition
health information
patient reported