Literature DB >> 29765290

Extended Half-Life Factor VIII and Factor IX Preparations.

Lukas Graf1.   

Abstract

In the last couple of years, several extended half-life factor VIII and factor IX preparations were intensively studied and gained approval. In order to extend half-lives, techniques like fusion to protein conjugates (Fc part of IgG1 or albumin), chemical modification (PEGylation), and protein sequence modification are implemented. With these techniques, it is possible to extend half-lives of factor IX products 4- to 6- fold, while half-life extension of factor VIII products is limited to 1.5- to 2-fold due to their interaction with von Willebrand factor. Nevertheless, both extended half-life factor VIII and IX products have improved and facilitated prophylactic factor replacement therapy in hemophilia A and B, respectively. Extended half-life factor concentrates pose challenges to coagulation laboratories because accurate therapy monitoring is not possible with all factor activity assays currently used.

Entities:  

Keywords:  Extended half-life; Factor concentrates; Hemophilia A and B; Prophylaxis

Year:  2018        PMID: 29765290      PMCID: PMC5939656          DOI: 10.1159/000488060

Source DB:  PubMed          Journal:  Transfus Med Hemother        ISSN: 1660-3796            Impact factor:   3.747


  46 in total

1.  Comparative pharmacokinetics of rVIII-SingleChain and octocog alfa (Advate(®) ) in patients with severe haemophilia A.

Authors:  R Klamroth; M Simpson; M von Depka-Prondzinski; J C Gill; M Morfini; J S Powell; E Santagostino; J Davis; A Huth-Kühne; C Leissinger; P Neumeister; D Bensen-Kennedy; A Feussner; T Limsakun; M Zhou; A Veldman; K St Ledger; N Blackman; I Pabinger
Journal:  Haemophilia       Date:  2016-07-19       Impact factor: 4.287

2.  Recommendations for the use of albumin and immunoglobulins.

Authors:  Giancarlo Maria Liumbruno; Francesco Bennardello; Angela Lattanzio; Pierluigi Piccoli; Gina Rossettias
Journal:  Blood Transfus       Date:  2009-07       Impact factor: 3.443

Review 3.  Life in the shadow of a dominant partner: the FVIII-VWF association and its clinical implications for hemophilia A.

Authors:  Steven W Pipe; Robert R Montgomery; Kathleen P Pratt; Peter J Lenting; David Lillicrap
Journal:  Blood       Date:  2016-09-01       Impact factor: 22.113

Review 4.  Longer-acting clotting factor concentrates for hemophilia.

Authors:  J S Powell
Journal:  J Thromb Haemost       Date:  2015-06       Impact factor: 5.824

Review 5.  PEGylation, successful approach to drug delivery.

Authors:  Francesco M Veronese; Gianfranco Pasut
Journal:  Drug Discov Today       Date:  2005-11-01       Impact factor: 7.851

6.  Haemophilia patients' unmet needs and their expectations of the new extended half-life factor concentrates.

Authors:  S von Mackensen; W Kalnins; J Krucker; J Weiss; W Miesbach; M Albisetti; I Pabinger; J Oldenburg
Journal:  Haemophilia       Date:  2017-03-30       Impact factor: 4.287

Review 7.  Haemophilias A and B.

Authors:  Paula H B Bolton-Maggs; K John Pasi
Journal:  Lancet       Date:  2003-05-24       Impact factor: 79.321

Review 8.  Novel approaches to hemophilia therapy: successes and challenges.

Authors:  Valder R Arruda; Bhavya S Doshi; Benjamin J Samelson-Jones
Journal:  Blood       Date:  2017-10-10       Impact factor: 22.113

9.  Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A.

Authors:  Johnny Mahlangu; Jerry S Powell; Margaret V Ragni; Pratima Chowdary; Neil C Josephson; Ingrid Pabinger; Hideji Hanabusa; Naresh Gupta; Roshni Kulkarni; Patrick Fogarty; David Perry; Amy Shapiro; K John Pasi; Shashikant Apte; Ivan Nestorov; Haiyan Jiang; Shuanglian Li; Srividya Neelakantan; Lynda M Cristiano; Jaya Goyal; Jurg M Sommer; Jennifer A Dumont; Nigel Dodd; Karen Nugent; Gloria Vigliani; Alvin Luk; Aoife Brennan; Glenn F Pierce
Journal:  Blood       Date:  2013-11-13       Impact factor: 22.113

10.  Long-acting recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in children. Results of a phase 3 trial.

Authors:  Gili Kenet; Hervé Chambost; Christoph Male; Thierry Lambert; Susan Halimeh; Tatiana Chernova; Maria Elisa Mancuso; Julie Curtin; Christine Voigt; Yanyan Li; Iris Jacobs; Elena Santagostino
Journal:  Thromb Haemost       Date:  2016-09-01       Impact factor: 5.249

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  16 in total

1.  Subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors: phase 2 trial results.

Authors:  Amy D Shapiro; Pantep Angchaisuksiri; Jan Astermark; Gary Benson; Giancarlo Castaman; Pratima Chowdary; Hermann Eichler; Victor Jiménez-Yuste; Kaan Kavakli; Tadashi Matsushita; Lone Hvitfeldt Poulsen; Allison P Wheeler; Guy Young; Silva Zupancic-Salek; Johannes Oldenburg
Journal:  Blood       Date:  2019-11-28       Impact factor: 22.113

Review 2.  A Molecular Revolution in the Treatment of Hemophilia.

Authors:  John S S Butterfield; Kerry M Hege; Roland W Herzog; Radoslaw Kaczmarek
Journal:  Mol Ther       Date:  2019-11-13       Impact factor: 11.454

3.  Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9.

Authors:  Calvin J Stephens; Elvin J Lauron; Elena Kashentseva; Zhi Hong Lu; Wayne M Yokoyama; David T Curiel
Journal:  J Control Release       Date:  2019-02-13       Impact factor: 9.776

4.  Evaluation of pre-analytic heat treatment protocol used in the CDC Nijmegen-Bethesda assay for heat inactivation of extended half-life haemophilia treatment products.

Authors:  Amanda B Payne; Dorothy Ellingsen; Jennifer Driggers; Christopher J Bean; Connie H Miller
Journal:  Haemophilia       Date:  2019-12-06       Impact factor: 4.287

5.  Postmarketing safety and effectiveness of recombinant factor IX (nonacog alfa) in Japanese patients with haemophilia B.

Authors:  Katsuyuki Fukutake; Masashi Taki; Tadashi Matsushita; Michio Sakai; Ami Takata; Hiromi Yamaguchi; Toshiyuki Karumori
Journal:  Haemophilia       Date:  2019-06-06       Impact factor: 4.287

6.  Safety and effectiveness of Rixubis in patients with hemophilia B: a real-world, prospective, postmarketing surveillance study in South Korea.

Authors:  Eun Jin Choi; Tai Ju Hwang; Yong Mook Choi; Hugh Chul Kim; Myung Chul Yoo; Haylee Song; Kayode Badejo
Journal:  Blood Res       Date:  2020-12-31

7.  Sugar Matters: Improving In Vivo Clearance Rate of Highly Glycosylated Recombinant Plasma Proteins for Therapeutic Use.

Authors:  Sacha Zeerleder; Ruchira Engel; Tao Zhang; Dorina Roem; Gerard van Mierlo; Ineke Wagenaar-Bos; Sija Marieke van Ham; Manfred Wuhrer; Diana Wouters; Ilse Jongerius
Journal:  Pharmaceuticals (Basel)       Date:  2021-01-11

8.  Bleeding outcomes and factor utilization after switching to an extended half-life product for prophylaxis in haemophilia A in Austria.

Authors:  Cihan Ay; Clemens Feistritzer; Joachim Rettl; Gerhard Schuster; Anna Vavrovsky; Leonard Perschy; Ingrid Pabinger
Journal:  Sci Rep       Date:  2021-06-21       Impact factor: 4.379

9.  PROTECT VIII Kids: BAY 94-9027 (PEGylated Recombinant Factor VIII) safety and efficacy in previously treated children with severe haemophilia A.

Authors:  Elena Santagostino; Gili Kenet; Kathelijn Fischer; Tina Biss; Sanjay Ahuja; MacGregor Steele
Journal:  Haemophilia       Date:  2020-03-25       Impact factor: 4.287

Review 10.  Population Pharmacokinetics of Clotting Factor Concentrates and Desmopressin in Hemophilia.

Authors:  Tim Preijers; Lisette M Schütte; Marieke J H A Kruip; Marjon H Cnossen; Frank W G Leebeek; Reinier M van Hest; Ron A A Mathôt
Journal:  Clin Pharmacokinet       Date:  2021-01       Impact factor: 6.447

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