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

Pratima Chowdary

Thrombosis research 2020 Dec

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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.

Citation

Pratima Chowdary. Extended half-life recombinant products in haemophilia clinical practice - Expectations, opportunities and challenges. Thrombosis research. 2020 Dec;196:609-617