Pharmacokinetics and the transition to extended half-life factor concentrates: communication from the SSC of the ISTH

Journal of Thrombosis and Haemostasis : JTH
M V RagniSubcommittee on Factor VIII, Factor IX, and Rare Bleeding Disorders

Abstract

Extended half-life proteins (EHL) are increasingly used in clinical practice, but there is no standardized approach to sampling, interpretation and implementation of pharmacokinetics (PK) data to maximize treatment benefit. The goal of EHL treatment is to attain a trough level sufficient to protect against spontaneous bleeds and reduce infusion frequency and limitations on individual activity and lifestyle. Performing classical PK assessments requires multiple blood samples, which is burdensome for patients and providers. Herein we review a population pharmacokinetic (popPK) approach to estimate individual PK parameters to transition patients from standard half-life (SHL) to EHL concentrates. We propose that a minimum of two to four post-infusion samples is sufficient to estimate individual PK profiles, with sufficient certainty to maintain factor levels above 1% and achieve bleed-free lifestyles. We also survey current PK use in patients transitioning to EHL, review key PK parameters and popPK models, and recommend an approach to using PK in patients initiating or switching to EHL.

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Citations

Sep 27, 2018·Pediatric Blood & Cancer·Courtney D Thornburg
Nov 20, 2019·Haemophilia : the Official Journal of the World Federation of Hemophilia·Alanna McEneny-KingStacy E Croteau
Feb 25, 2020·Haemophilia : the Official Journal of the World Federation of Hemophilia·Yasuko FuruichiMidori Shima
Apr 22, 2020·Haemophilia : the Official Journal of the World Federation of Hemophilia·Thomas SinegreAurélien Lebreton
Jul 1, 2020·Haemophilia : the Official Journal of the World Federation of Hemophilia·Peter L TurecekUNKNOWN iPATH study group
Jul 10, 2019·Expert Review of Hematology·Miguel EscobarClaude Negrier
Jul 12, 2019·Research and Practice in Thrombosis and Haemostasis·Jacky K YuUNKNOWN WAPPS co‐investigators
Oct 17, 2020·American Journal of Hematology·Stacy E CroteauAllison P Wheeler
Mar 2, 2021·Haemophilia : the Official Journal of the World Federation of Hemophilia·Kun HuangZhenping Chen
Mar 19, 2021·Research and Practice in Thrombosis and Haemostasis·Jerome TeitelAlfonso Iorio
May 21, 2021·Haemophilia : the Official Journal of the World Federation of Hemophilia·Kun HuangRunhui Wu
Jun 26, 2021·The Lancet. Haematology·Margaret V Ragni
Aug 15, 2021·Hematology/oncology Clinics of North America·Craig D SeamanMargaret V Ragni
Sep 9, 2021·Blood Advances·Tine M H J GoedhartUNKNOWN OPTI-CLOT study group and SYMPHONY consortium

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