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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.
Lentz, Steven R; Kavakli, Kaan; Klamroth, Robert; Misgav, Mudi; Nagao, Azusa; Tosetto, Alberto; Jørgensen, Pernille Juul; Zak, Marek; Nemes, Laszlo.
Afiliación
  • Lentz SR; Division of Hematology, Oncology, and Blood & Marrow Transplantation Department of Internal Medicine University of Iowa Carver College of Medicine Iowa City Iowa USA.
  • Kavakli K; Department of Haematology Ege University Faculty of Medicine Children's Hospital Bornova Izmir Turkey.
  • Klamroth R; Department for Internal Medicine Vascular Medicine and Haemostaseology Vivantes Klinikum im Friedrichshain Berlin Germany.
  • Misgav M; National Hemophilia Center Sheba Medical Center Tel Hashomer Israel.
  • Nagao A; Department of Blood Coagulation Ogikubo Hospital Tokyo Japan.
  • Tosetto A; Hemophilia and Thrombosis Center Hematology Department San Bortolo Hospital Vicenza Italy.
  • Jørgensen PJ; Biostatistics Biopharm Novo Nordisk A/S Bagsværd Denmark.
  • Zak M; Medical and Science Biopharm Novo Nordisk A/S Bagsværd Denmark.
  • Nemes L; National Hemophilia Center and Hemostasis Department Medical Center of the Hungarian Defence Forces Budapest Hungary.
Res Pract Thromb Haemost ; 6(2): e12674, 2022 Feb.
Article en En | MEDLINE | ID: mdl-35308099
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 (≈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.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Res Pract Thromb Haemost Año: 2022 Tipo del documento: Article Pais de publicación: Estados Unidos
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Res Pract Thromb Haemost Año: 2022 Tipo del documento: Article Pais de publicación: Estados Unidos