Clinical studies of extended-half-life recombinant FVIII products for prophylaxis in adults and children: A critical review from the physician's perspective

Cedric Hermans, Mark T. Reding, Jan Astermark, Robert Klamroth, Maria Elisa Mancuso

Research output: Contribution to journalReview articlepeer-review

7 Scopus citations

Abstract

This review compares the methodology of published clinical studies investigating the extended-half-life (EHL) factor VIII (FVIII) products, rFVIIIFc (efmoroctocog alfa, Elocta®/Eloctate®), BAY 94-9027 (damoctocog alfa pegol, Jivi®), BAX 855 (rurioctocog alfa pegol, Adynovate®) and N8-GP (turoctocog alfa pegol, Esperoct®) including the phase 2/3 studies, A-LONG (NCT01181128), PROTECT VIII (NCT01580293), PROLONG-ATE (NCT01736475) and pathfinder2 (NCT01480180), respectively, and their corresponding pediatric studies and extensions. Study results are interpreted from a treating physician's perspective, translating into evidence-based, real-life use of the different EHL recombinant FVIII products for personalized prophylaxis. The similarities between the studies include methodology, objectives, study design and cohort size. The differences include duration, prophylactic dosing intervals, number of patient arms, use of control group and randomization, and treatment allocation. Comparing these studies broadens physicians’ understanding of each treatment's applicability. Further evaluation of study data and future real-world studies should help physicians to confidently individualize and select treatment for each patient.

Original languageEnglish (US)
Article number103678
JournalCritical Reviews in Oncology/Hematology
Volume174
DOIs
StatePublished - Jun 2022

Bibliographical note

Funding Information:
This manuscript, PROTECT VIII, PROTECT VIII Kids and PROTECT VIII extension studies were funded by Bayer. Medical writing assistance was provided by Rachel Price and Frank Biegun of Darwin Health Communications (London, England) and was fully funded by Bayer.

Publisher Copyright:
© 2022 The Authors

Keywords

  • Extended-half-life
  • Factor VIII
  • Fc fusion
  • Hemophilia A
  • Polyethylene glycol
  • Prophylaxis

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