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

doi:10.1016/j.critrevonc.2022.103678

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

Medicine - Hematology, Oncology, Transplant

Research output: Contribution to journal › Review article › peer-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 language	English (US)
Article number	103678
Journal	Critical Reviews in Oncology/Hematology
Volume	174
DOIs	https://doi.org/10.1016/j.critrevonc.2022.103678
State	Published - 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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title = "Clinical studies of extended-half-life recombinant FVIII products for prophylaxis in adults and children: A critical review from the physician's perspective",

abstract = "This review compares the methodology of published clinical studies investigating the extended-half-life (EHL) factor VIII (FVIII) products, rFVIIIFc (efmoroctocog alfa, Elocta{\textregistered}/Eloctate{\textregistered}), BAY 94-9027 (damoctocog alfa pegol, Jivi{\textregistered}), BAX 855 (rurioctocog alfa pegol, Adynovate{\textregistered}) and N8-GP (turoctocog alfa pegol, Esperoct{\textregistered}) 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{\textquoteright} 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.",

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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: {\textcopyright} 2022 The Authors",

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AU - Mancuso, Maria Elisa

N1 - 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

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

Abstract

Bibliographical note

Keywords

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