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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

Hermans, Cedric ; Reding, Mark T. ; Astermark, Jan LU ; Klamroth, Robert and Mancuso, Maria Elisa (2022) In Critical Reviews in Oncology/Hematology 174.
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... (More)

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.

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author
; ; ; and
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
Extended-half-life, Factor VIII, Fc fusion, Hemophilia A, Polyethylene glycol, Prophylaxis
in
Critical Reviews in Oncology/Hematology
volume
174
article number
103678
publisher
Elsevier
external identifiers
  • scopus:85129515015
  • pmid:35390452
ISSN
1040-8428
DOI
10.1016/j.critrevonc.2022.103678
language
English
LU publication?
yes
id
9abe4610-4ea7-4e4c-b6f5-7013e2b32a96
date added to LUP
2022-08-15 13:12:23
date last changed
2024-06-27 19:40:34
@article{9abe4610-4ea7-4e4c-b6f5-7013e2b32a96,
  abstract     = {{<p>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.</p>}},
  author       = {{Hermans, Cedric and Reding, Mark T. and Astermark, Jan and Klamroth, Robert and Mancuso, Maria Elisa}},
  issn         = {{1040-8428}},
  keywords     = {{Extended-half-life; Factor VIII; Fc fusion; Hemophilia A; Polyethylene glycol; Prophylaxis}},
  language     = {{eng}},
  publisher    = {{Elsevier}},
  series       = {{Critical Reviews in Oncology/Hematology}},
  title        = {{Clinical studies of extended-half-life recombinant FVIII products for prophylaxis in adults and children : A critical review from the physician's perspective}},
  url          = {{http://dx.doi.org/10.1016/j.critrevonc.2022.103678}},
  doi          = {{10.1016/j.critrevonc.2022.103678}},
  volume       = {{174}},
  year         = {{2022}},
}