Development of a Plasminogen Population PK model supporting prophylactic replacement therapy for Plasminogen deficient patients within the WAPPS-Hemo platform.
hypoplasminogenemia
pharmacokinetics
plasminogen deficiency
Journal
Haemophilia : the official journal of the World Federation of Hemophilia
ISSN: 1365-2516
Titre abrégé: Haemophilia
Pays: England
ID NLM: 9442916
Informations de publication
Date de publication:
02 May 2024
02 May 2024
Historique:
revised:
10
04
2024
received:
26
01
2024
accepted:
12
04
2024
medline:
3
5
2024
pubmed:
3
5
2024
entrez:
3
5
2024
Statut:
aheadofprint
Résumé
Plasminogen deficiency is an ultra rare disease whose patients may develop ligneous lesions if untreated. Prophylactic replacement therapy with plasma derived plasminogen, Ryplazim, is efficient in treating lesions and could benefit from pharmacokinetic (PK) tailoring. The objectives of this study are to develop, evaluate and integrate into the WAPPS-Hemo platform a Population PK model supporting prophylactic replacement therapy for Plasminogen deficient patients. Population PK modelling and evaluations followed the same protocol performed for factor VIII and IX concentrates. Limited sampling analysis used dosing and sampling scenarios in accordance with recommended treatment for Ryplazim. The population PK model, derived from 16 participants included in previous clinical studies, was a 2-compartment model whose variability was best described by fat-free mass. Evaluations showed that the model described well the data and Bayesian forecasting in limited sampling environment led to acceptable precision for PK parameters relevant to plasminogen treatment. The model was integrated into the WAPPS-Hemo webservice to help individualize prophylactic treatment in plasminogen deficient patients. Prospective PK data to be collected through the WAPPS-Hemo database will be used to better understand plasminogen PK and improve patient care.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Informations de copyright
© 2024 The Authors. Haemophilia published by John Wiley & Sons Ltd.
Références
Schuster V, Seregard S. Ligneous conjunctivitis. Surv Ophthalmol. 2003;48(4):369‐388.
Tait R, Walker ID, Conkie JA, Islam SI, McCall F. Isolated familial plasminogen deficiency may not be a risk factor for thrombosis. Thromb Haemost. 1996;76(6):1004‐1008.
Mehta R, Shapiro AD. Plasminogen deficiency. Haemophilia. 2008;14(6):1261‐1268.
Shapiro AD, Menegatti M, Palla R, et al. An international registry of patients with plasminogen deficiency (HISTORY). Haematologica. 2020;105(3):554‐561.
Schuster V, Hügle B, Tefs K. Plasminogen deficiency. J Thromb Haemost. 2007;5(12):2315‐2322.
Shapiro AD, Nakar C, Parker JM, Thibaudeau K, Crea R, Sandset PM. Plasminogen, human‐tvmh for the treatment of children and adults with plasminogen deficiency type 1. Haemophilia. 2023;29(6):1556‐1564.
U.S. Food & Drug Administration. RYPLAZIM. 2022; Available from: https://www.fda.gov/vaccines‐blood‐biologics/ryplazim
Shapiro AD, Nakar C, Parker JM, et al. Plasminogen replacement therapy for the treatment of children and adults with congenital plasminogen deficiency. Blood. 2018;131(12):1301‐1310.
Iorio A, Keepanasseril A, Foster G, et al, WAPPS‐Hemo co‐investigator network. 2016. Development of a Web‐Accessible Population Pharmacokinetic Service‐Hemophilia (WAPPS‐Hemo): study protocol. JMIR Res Protoc. 2016;5(4):e239.
Al‐Sallami H, Goulding A, Grant A, Taylor R, Holford N, Duffull SB. Prediction of fat‐free mass in children. Clin Pharmacokinet. 2015;54(11):1169‐1178.
Hajducek D, Chelle P, Hermans C, et al. Development and evaluation of the population pharmacokinetic models for FVIII and FIX concentrates of the WAPPS‐Hemo project. Haemophilia. 2020;0:1‐17.
Mceneny‐King A, Foster G, Iorio A, Edginton AN. Data analysis protocol for the development and evaluation of population pharmacokinetic models for incorporation into the Web‐Accessible Population Pharmacokinetic Service—Hemophilia (WAPPS‐Hemo). JMIR Res Protoc. 2016;5(4):e232.
Beal S, Boeckmann A, Sheiner L. NONMEM Users Guide. Parts I‐VIII ICON Development Solutions. ICON.
R Foundation for Statistical Computing, R: A Language and Environment for Statistical Computing; 2023.
Wickham H. ggplot2: Elegant Graphics for Data Analysis. Springer‐Verlag New York; 2016.
Savic RM, Karlsson MO. Importance of shrinkage in empirical bayes estimates for diagnostics: problems and solutions. AAPS J. 2009;11(3):558‐569.
Bergstrand M, Hooker AC, Wallin JE, Karlsson MO. Prediction‐corrected visual predictive checks for diagnosing nonlinear mixed‐effects models. AAPS J. 2011;13(2):143‐151.
Collen D, Tytgat G, Claeys H, Verstraete M, Wallén P. Metabolism of plasminogen in healthy subjects: effect of tranexamic acid. J Clin Investig. 1972;51(6):1310‐1318.
Brekkan A, Berntorp E, Jensen K, Nielsen EI, Jönsson S. Population pharmacokinetics of plasma‐derived factor IX: procedures for dose individualization. J Thromb Haemost. 2016;14:724‐732.
McEneny‐King A, Chelle P, Goggans MH, et al. Limited sampling strategies for accurate determination of extended half‐life factor VIII pharmacokinetics in severe haemophilia A patients. Haemophilia. 2021;0:1‐9.