Literature DB >> 31282771

Switching patients in the age of long-acting recombinant products?

Miguel Escobar1, Elena Santagostino2, Maria Elisa Mancuso2, Michiel Coppens3, Vinod Balasa4, Jason A Taylor5, Alfonso Iorio6, Claude Negrier7.   

Abstract

Introduction: Prophylaxis with factor replacement therapy is the gold standard for the treatment of hemophilia, but this often requires frequent infusions. A number of long-acting factor products have been developed to reduce the burden on patients. Areas covered: This is an overview of information presented at two symposia held at the World Federation of Hemophilia and International Society on Thrombosis and Haemostasis - Scientific and Standardization Committee annual meetings. The pharmacokinetic, safety and efficacy data for long-acting recombinant products are reviewed, with a focus on recombinant factor IX albumin fusion protein (rIX-FP) and rVIII-SingleChain. This overview also provides a guide for managing a patient's switch to long-acting products. Expert opinion: Long-acting products may allow patients to maintain or decrease bleeding rates whilst increasing their dosing interval, which may in turn reduce the burden on patients and caregivers. When switching patients to long-acting products health-care professionals should provide balanced and thorough education to the patient, whilst supporting their emotional well-being. Regimens should address patients' needs and goals but should also be guided by clinical phenotype and pharmacokinetic assessment. Follow-up should assess safety concerns, bleeding rates, joint health and the impact of the regimen on patients' lifestyle.

Entities:  

Keywords:  Hemophilia; meeting report; personalized treatment; pharmacokinetics

Mesh:

Substances:

Year:  2019        PMID: 31282771     DOI: 10.1080/17474086.2018.1564032

Source DB:  PubMed          Journal:  Expert Rev Hematol        ISSN: 1747-4094            Impact factor:   2.929


  5 in total

1.  Clinical, humanistic, and economic burden of severe hemophilia B in the United States: Results from the CHESS US and CHESS US+ population surveys.

Authors:  Tom Burke; Sohaib Asghar; Jamie O'Hara; Eileen K Sawyer; Nanxin Li
Journal:  Orphanet J Rare Dis       Date:  2021-03-20       Impact factor: 4.123

Review 2.  IDELVION: A Comprehensive Review of Clinical Trial and Real-World Data.

Authors:  Miguel Escobar; Maria Elisa Mancuso; Cedric Hermans; Cindy Leissinger; Wilfried Seifert; Yanyan Li; William McKeand; Johannes Oldenburg
Journal:  J Clin Med       Date:  2022-02-18       Impact factor: 4.241

3.  Current Choices and Management of Treatment in Persons with Severe Hemophilia A without Inhibitors: A Mini-Delphi Consensus.

Authors:  Antonio Coppola; Massimo Franchini; Giovanni Pappagallo; Alessandra Borchiellini; Raimondo De Cristofaro; Angelo Claudio Molinari; Rita Carlotta Santoro; Cristina Santoro; Annarita Tagliaferri
Journal:  J Clin Med       Date:  2022-02-02       Impact factor: 4.241

4.  Efficacy and Safety of Weekly Somatrogon vs Daily Somatropin in Children With Growth Hormone Deficiency: A Phase 3 Study.

Authors:  Cheri L Deal; Joel Steelman; Elpis Vlachopapadopoulou; Renata Stawerska; Lawrence A Silverman; Moshe Phillip; Ho-Seong Kim; CheolWoo Ko; Oleg Malievskiy; Jose F Cara; Carl L Roland; Carrie Turich Taylor; Srinivas Rao Valluri; Michael P Wajnrajch; Aleksandra Pastrak; Bradley S Miller
Journal:  J Clin Endocrinol Metab       Date:  2022-06-16       Impact factor: 6.134

5.  Matching-Adjusted Indirect Comparison of Efficacy and Consumption of rVIII-SingleChain Versus Two Recombinant FVIII Products Used for Prophylactic Treatment of Adults/Adolescents with Severe Haemophilia A.

Authors:  Santiago Bonanad; Ramiro Núñez; Jose Luis Poveda; Karin Kurnik; Georg Goldmann; Valeska Andreozzi; Björn Vandewalle; Sandra Santos
Journal:  Adv Ther       Date:  2021-08-08       Impact factor: 3.845

  5 in total

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