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

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
Article number103678
JournalCritical Reviews in Oncology/Hematology
Volume174
DOIs
Publication statusPublished - 2022 Jun

Subject classification (UKÄ)

  • Health Care Service and Management, Health Policy and Services and Health Economy

Free keywords

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

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