Turoctocog alfa pegol (N8‐GP) in severe hemophilia A: Long‐term safety and efficacy in previously treated patients of all ages in the pathfinder8 study

Steven R. Lentz; Kaan Kavakli; Robert Klamroth; Mudi Misgav; Azusa Nagao; Alberto Tosetto; Pernille Juul Jørgensen; Marek Zak; Laszlo Nemes

doi:10.1002/rth2.12674

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Turoctocog alfa pegol (N8‐GP) in severe hemophilia A: Long‐term safety and efficacy in previously treated patients of all ages in the pathfinder8 study

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Turoctocog alfa pegol (N8‐GP) in severe hemophilia A: Long‐term safety and efficacy in previously treated patients of all ages in the pathfinder8 study

Steven R. Lentz, Kaan Kavakli, Robert Klamroth, Mudi Misgav, Azusa Nagao, Alberto Tosetto, Pernille Juul Jørgensen, Marek Zak and Laszlo Nemes

Research and practice in thrombosis and haemostasis, Vol.6(2), pp.e12674-e12674

02/2022

DOI: 10.1002/rth2.12674

PMCID: PMC8918113

PMID: 35308099

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https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8918113View

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Abstract

N8‐GP (turoctocog alfa pegol; Esperoct) is a glycoPEGylated human recombinant factor VIII (FVIII). Pathfinder8 (NCT01480180) was a phase 3, multinational, open‐label, nonrandomized trial to investigate the long‐term safety and efficacy of N8‐GP in people of all ages with severe hemophilia A previously treated with N8‐GP. Patients were recruited from the completed phase 3 pathfinder2 and pathfinder5 trials to receive intravenous N8‐GP prophylaxis for up to 104 weeks, administered every 7 days, twice weekly, or three times weekly. Primary and secondary end points were the number of adverse events (AEs) reported and efficacy of treatment, respectively. Overall, 160 patients were exposed to N8‐GP for a mean of 179 exposure days and 681 calendar days (≈1.9 years) per patient. In total, 119 patients experienced 510 AEs, corresponding to a rate of 1.71 AEs per patient‐year of exposure; 97.5% of AEs were mild or moderate in severity, and no AEs led to withdrawal. No patients developed FVIII inhibitors during the trial. The Poisson estimate of mean annualized bleeding rate for all bleeds (excluding surgery) and across all regimens was 1.10 (median, 0.00), and for spontaneous bleeds was 0.61 (median, 0.00). Most (55.6%) patients experienced no bleeds that required FVIII treatment (excluding perioperative bleeds). The estimated hemostatic success rate for the treatment of 322 bleeding episodes (excluding surgery) was 95.8%, including missing values as failure. Long‐term prophylactic use of N8‐GP appeared safe and efficacious across all age groups in people with severe hemophilia A previously treated with N8‐GP.

factor VIII

hemophilia A

prophylaxis

safety

turoctocog alfa pegol

Details

Title: Subtitle: Turoctocog alfa pegol (N8‐GP) in severe hemophilia A: Long‐term safety and efficacy in previously treated patients of all ages in the pathfinder8 study
Creators: Steven R. Lentz - Roy J. and Lucille A. Carver College of Medicine
Kaan Kavakli - Ege University
Robert Klamroth - Klinikum im Friedrichshain
Mudi Misgav - Sheba Medical Center
Azusa Nagao - Ogikubo Hospital
Alberto Tosetto - Ospedale San Bortolo
Pernille Juul Jørgensen - Biostatistics Biopharm, Novo Nordisk A/S, Bagsværd, Denmark
Marek Zak - Novo Nordisk (Denmark)
Laszlo Nemes - National Hemophilia Center and Hemostasis Department Medical Center of the Hungarian Defence Forces Budapest Hungary.
Resource Type: Journal article
Publication Details: Research and practice in thrombosis and haemostasis, Vol.6(2), pp.e12674-e12674
DOI: 10.1002/rth2.12674
PMID: 35308099
PMCID: PMC8918113
NLM abbreviation: Res Pract Thromb Haemost
ISSN: 2475-0379
eISSN: 2475-0379
Publisher: Elsevier Inc
Grant note: DOI: 10.13039/501100004191, name: Novo Nordisk
Language: English
Date published: 02/2022
Academic Unit: Hematology, Oncology, and Blood & Marrow Transplantation; Internal Medicine
Record Identifier: 9984359849602771

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