PMID- 35308099
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20230328
IS  - 2475-0379 (Electronic)
IS  - 2475-0379 (Linking)
VI  - 6
IP  - 2
DP  - 2022 Feb
TI  - 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.
PG  - e12674
LID - 10.1002/rth2.12674 [doi]
LID - e12674
AB  - BACKGROUND: N8-GP (turoctocog alfa pegol; Esperoct) is a glycoPEGylated human 
      recombinant factor VIII (FVIII). OBJECTIVES: 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/METHOD: 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. RESULTS: Overall, 160 patients were exposed to N8-GP for a mean of 
      179 exposure days and 681 calendar days ( approximately 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. CONCLUSIONS: 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.
CI  - (c) 2022 The Authors. Research and Practice in Thrombosis and Haemostasis published 
      by Wiley Periodicals LLC on behalf of International Society on Thrombosis and 
      Haemostasis (ISTH).
FAU - Lentz, Steven R
AU  - Lentz SR
AUID- ORCID: 0000-0002-8885-4718
AD  - Division of Hematology, Oncology, and Blood & Marrow Transplantation Department 
      of Internal Medicine University of Iowa Carver College of Medicine Iowa City Iowa 
      USA.
FAU - Kavakli, Kaan
AU  - Kavakli K
AD  - Department of Haematology Ege University Faculty of Medicine Children's Hospital 
      Bornova Izmir Turkey.
FAU - Klamroth, Robert
AU  - Klamroth R
AD  - Department for Internal Medicine Vascular Medicine and Haemostaseology Vivantes 
      Klinikum im Friedrichshain Berlin Germany.
FAU - Misgav, Mudi
AU  - Misgav M
AD  - National Hemophilia Center Sheba Medical Center Tel Hashomer Israel.
FAU - Nagao, Azusa
AU  - Nagao A
AD  - Department of Blood Coagulation Ogikubo Hospital Tokyo Japan.
FAU - Tosetto, Alberto
AU  - Tosetto A
AUID- ORCID: 0000-0002-0119-5204
AD  - Hemophilia and Thrombosis Center Hematology Department San Bortolo Hospital 
      Vicenza Italy.
FAU - Jorgensen, Pernille Juul
AU  - Jorgensen PJ
AD  - Biostatistics Biopharm Novo Nordisk A/S Bagsvaerd Denmark.
FAU - Zak, Marek
AU  - Zak M
AD  - Medical and Science Biopharm Novo Nordisk A/S Bagsvaerd Denmark.
FAU - Nemes, Laszlo
AU  - Nemes L
AD  - National Hemophilia Center and Hemostasis Department Medical Center of the 
      Hungarian Defence Forces Budapest Hungary.
LA  - eng
PT  - Journal Article
DEP - 20220312
PL  - United States
TA  - Res Pract Thromb Haemost
JT  - Research and practice in thrombosis and haemostasis
JID - 101703775
PMC - PMC8918113
OTO - NOTNLM
OT  - factor VIII
OT  - hemophilia A
OT  - prophylaxis
OT  - safety
OT  - turoctocog alfa pegol
EDAT- 2022/03/22 06:00
MHDA- 2022/03/22 06:01
PMCR- 2022/03/12
CRDT- 2022/03/21 08:35
PHST- 2021/11/08 00:00 [received]
PHST- 2022/01/13 00:00 [revised]
PHST- 2022/01/17 00:00 [accepted]
PHST- 2022/03/21 08:35 [entrez]
PHST- 2022/03/22 06:00 [pubmed]
PHST- 2022/03/22 06:01 [medline]
PHST- 2022/03/12 00:00 [pmc-release]
AID - S2475-0379(22)01165-7 [pii]
AID - RTH212674 [pii]
AID - 10.1002/rth2.12674 [doi]
PST - epublish
SO  - Res Pract Thromb Haemost. 2022 Mar 12;6(2):e12674. doi: 10.1002/rth2.12674. 
      eCollection 2022 Feb.