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

Miguel Escobar, Elena Santagostino, Maria Elisa Mancuso, Michiel Coppens, Vinod Balasa, Jason Taylor, Alfonso Iorio, Claude Negrier

Research output: Contribution to journalArticle

1 Citation (Scopus)

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.

Original languageEnglish (US)
Pages (from-to)1-13
Number of pages13
JournalExpert Review of Hematology
Volume12
Issue numbersup1
DOIs
StatePublished - Jan 14 2019

Fingerprint

Pharmacokinetics
Hemorrhage
Safety
Factor IX
Expert Testimony
Hemophilia A
Patient Education
Caregivers
Life Style
Albumins
Thrombosis
Joints
Delivery of Health Care
Phenotype
Health
Therapeutics
Proteins

Keywords

  • Hemophilia
  • meeting report
  • personalized treatment
  • pharmacokinetics

ASJC Scopus subject areas

  • Hematology

Cite this

Escobar, M., Santagostino, E., Mancuso, M. E., Coppens, M., Balasa, V., Taylor, J., ... Negrier, C. (2019). Switching patients in the age of long-acting recombinant products? Expert Review of Hematology, 12(sup1), 1-13. https://doi.org/10.1080/17474086.2018.1564032

Switching patients in the age of long-acting recombinant products? / Escobar, Miguel; Santagostino, Elena; Mancuso, Maria Elisa; Coppens, Michiel; Balasa, Vinod; Taylor, Jason; Iorio, Alfonso; Negrier, Claude.

In: Expert Review of Hematology, Vol. 12, No. sup1, 14.01.2019, p. 1-13.

Research output: Contribution to journalArticle

Escobar, M, Santagostino, E, Mancuso, ME, Coppens, M, Balasa, V, Taylor, J, Iorio, A & Negrier, C 2019, 'Switching patients in the age of long-acting recombinant products?', Expert Review of Hematology, vol. 12, no. sup1, pp. 1-13. https://doi.org/10.1080/17474086.2018.1564032
Escobar, Miguel ; Santagostino, Elena ; Mancuso, Maria Elisa ; Coppens, Michiel ; Balasa, Vinod ; Taylor, Jason ; Iorio, Alfonso ; Negrier, Claude. / Switching patients in the age of long-acting recombinant products?. In: Expert Review of Hematology. 2019 ; Vol. 12, No. sup1. pp. 1-13.
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