TY - JOUR
T1 - Clinical studies of extended-half-life recombinant FVIII products for prophylaxis in adults and children
T2 - A critical review from the physician's perspective
AU - Hermans, Cedric
AU - Reding, Mark T.
AU - Astermark, Jan
AU - Klamroth, Robert
AU - Mancuso, Maria Elisa
PY - 2022/6
Y1 - 2022/6
N2 - 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.
AB - 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.
KW - Extended-half-life
KW - Factor VIII
KW - Fc fusion
KW - Hemophilia A
KW - Polyethylene glycol
KW - Prophylaxis
U2 - 10.1016/j.critrevonc.2022.103678
DO - 10.1016/j.critrevonc.2022.103678
M3 - Review article
C2 - 35390452
AN - SCOPUS:85129515015
VL - 174
JO - Critical Reviews in Oncology/Hematology
JF - Critical Reviews in Oncology/Hematology
SN - 1040-8428
M1 - 103678
ER -