Rivaroxaban for treatment of pediatric venous thromboembolism. An Einstein-Jr phase 3 dose-exposure-response evaluation

Background Recently, the randomized EINSTEIN-Jr study showed similar efficacy and safety for rivaroxaban and standard anticoagulation for treatment of pediatric venous thromboembolism (VTE). The rivaroxaban dosing strategy was established based on phase 1 and 2 data in children and through pharmacok...

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Detalles Bibliográficos
Autores: Young, G, Lensing, AWA, Monagle, P, Male, C, Thelen, K, Willmann, S, Palumbo, JS, Kumar, R, Nurmeev, I, Hege, K, Bajolle, F, Connor, P, Hooimeijer, HL, Torres, M, Chan, AKC, Kenet, G, Holzhauer, S, Santamaria, A, Amedro, P, Beyer-Westendorf, J, Martinelli, I, Massicotte, MP, Smith, WT, Berkowitz, SD, Schmidt, S, Price, V, Prins, MH, Kubitza, D, EINSTEIN-Jr Phase 3 Investigators
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2020
País:España
Institución:Fundación para el Fomento de la Investigación Sanitaria y Biomédica de la Comunitat Valenciana (FISABIO)
Repositorio:r-FISABIO. Repositorio Institucional de Producción Científica
OAI Identifier:oai:fisabio.fundanetsuite.com:p13228
Acceso en línea:https://fisabio.portalinvestigacion.com/publicaciones/13228
Access Level:acceso abierto
Palabra clave:anticoagulation
bodyweight-adjusted dosing
pediatric patients
pharmacokinetics
rivaroxaban
suspension
venous thromboembolism
Descripción
Sumario:Background Recently, the randomized EINSTEIN-Jr study showed similar efficacy and safety for rivaroxaban and standard anticoagulation for treatment of pediatric venous thromboembolism (VTE). The rivaroxaban dosing strategy was established based on phase 1 and 2 data in children and through pharmacokinetic (PK) modeling. Methods Rivaroxaban treatment with tablets or the newly developed granules-for-oral suspension formulation was bodyweight-adjusted and administered once-daily, twice-daily, or thrice-daily for children with bodyweights of >= 30, >= 12 to <30, and <12 kg, respectively. Previously, these regimens were confirmed for children weighing >= 20 kg but only predicted in those <20 kg. Based on sparse blood sampling, the daily area under the plasma concentration-time curve [AUC((0-24)ss)] and trough [C-trough,C-ss] and maximum [C-max,C-ss] steady-state plasma concentrations were derived using population PK modeling. Exposure-response graphs were generated to evaluate the potential relationship of individual PK parameters with recurrent VTE, repeat imaging outcomes, and bleeding or adverse events. A taste-and-texture questionnaire was collected for suspension-recipients. Results Of the 335 children (aged 0-17 years) allocated to rivaroxaban, 316 (94.3%) were evaluable for PK analyses. Rivaroxaban exposures were within the adult exposure range. No clustering was observed for any of the PK parameters with efficacy, bleeding, or adverse event outcomes. Results were similar for the tablet and suspension formulation. Acceptability and palatability of the suspension were favorable. Discussion Based on this analysis and the recently documented similar efficacy and safety of rivaroxaban compared with standard anticoagulation, we conclude that bodyweight-adjusted pediatric rivaroxaban regimens with either tablets or suspension are validated and provide for appropriate treatment of children with VTE.