Literature DB >> 31883700

Extended half-life recombinant products in haemophilia clinical practice - Expectations, opportunities and challenges.

Pratima Chowdary1.   

Abstract

Extended half-life (EHL) products have shown robust efficacy in clinical trials, whilst allowing for less intense treatment regimens when compared with standard half-life products. Regimen optimisation with EHL products could lead to further improvements in bleeding rates, quality of life and reductions in treatment burden. Patients now expect good efficacy, a lower treatment burden and equivalent safety when compared with standard half-life products. As our knowledge base grows these expectations have evolved and targeting an annualised bleeding rate of zero has become a more realistic clinical goal. Personalised prophylaxis can help patients achieve these goals. However, a number of challenges still remain, including cost, challenges in predicting outcomes for patients and differences in patients' and clinicians' expectations. When switching a patient, comprehensive patient care can reduce the impact of these issues. This review presents in brief the protein therapeutics with an extended half-life, including key trial results, challenges of chronic care that impact on patients' outcomes and how the modified proteins might help address some of these issues. In addition, practical steps for managing the switch to EHL products are presented.
Copyright © 2019 Elsevier Ltd. All rights reserved.

Entities:  

Keywords:  Efficacy; Factor IX; Factor VIII; Personalised prophylaxis; Switching; Treatment burden

Mesh:

Substances:

Year:  2019        PMID: 31883700     DOI: 10.1016/j.thromres.2019.12.012

Source DB:  PubMed          Journal:  Thromb Res        ISSN: 0049-3848            Impact factor:   3.944


  7 in total

1.  Predictive Modeling Identifies Total Bleeds at 12-Weeks Postswitch to N8-GP Prophylaxis as a Predictor of Treatment Response.

Authors:  Pratima Chowdary; Kingsley Hampton; Victor Jiménez-Yuste; Guy Young; Soraya Benchikh El Fegoun; Aidan Cooper; Erik Scalfaro; Andreas Tiede
Journal:  Thromb Haemost       Date:  2021-12-05       Impact factor: 6.681

2.  Twelve-month prevalence of haemarthrosis and joint disease using the Haemophilia Joint Health score: evaluation of the UK National Haemophilia Database and Haemtrack patient reported data: an observational study.

Authors:  Richard A Wilkins; David Stephensen; Heidi Siddle; Martin J Scott; Hua Xiang; Elizabeth Horn; Ben Palmer; Graham J Chapman; Michael Richards; Rebecca Walwyn; Anthony Redmond
Journal:  BMJ Open       Date:  2022-01-12       Impact factor: 2.692

3.  Illustrative Cases from the Pathfinder Clinical Trials of Patients with Hemophilia A Treated with Turoctocog Alfa Pegol (N8-GP).

Authors:  Robert Klamroth; Kingsley Hampton; Sonata Saulyte Trakymienė; Lars Korsholm; Manuel Carcao
Journal:  Patient Prefer Adherence       Date:  2021-11-04       Impact factor: 2.711

4.  Turoctocog alfa pegol (N8-GP) in severe hemophilia A: Long-term safety and efficacy in previously treated patients of all ages in the pathfinder8 study.

Authors:  Steven R Lentz; Kaan Kavakli; Robert Klamroth; Mudi Misgav; Azusa Nagao; Alberto Tosetto; Pernille Juul Jørgensen; Marek Zak; Laszlo Nemes
Journal:  Res Pract Thromb Haemost       Date:  2022-03-12

5.  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

6.  Impact of the COVID-19 pandemic on therapeutic choices in thrombosis-hemostasis.

Authors:  Cedric Hermans; Catherine Lambert
Journal:  J Thromb Haemost       Date:  2020-05-11       Impact factor: 5.824

Review 7.  An Update on Laboratory Diagnostics in Haemophilia A and B.

Authors:  Jens Müller; Wolfgang Miesbach; Florian Prüller; Thomas Siegemund; Ute Scholz; Ulrich J Sachs
Journal:  Hamostaseologie       Date:  2022-02-01       Impact factor: 2.145

  7 in total

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