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The safety and efficacy of N8-GP (turoctocog alfa pegol) in previously untreated pediatric patients with hemophilia A.

ABSTRACT: N8-GP (turoctocog alfa pegol) is a recombinant, glycoPEGylated, extended half-life, factor VIII replacement product. Here, we examined the immunogenicity, safety, and efficacy of N8-GP in previously untreated patients (PUPs). pathfinder6 is an ongoing, open-label, phase 3 trial that enrolled PUPs with severe hemophilia A and were aged <6 years. The primary end point was the incidence of factor VIII inhibitors (≥0.6 Bethesda units [BU]). Eighty patients received ≥1 N8-GP dose and were included in this analysis; ≥50 patients had ≥50 exposure days to N8-GP. The inhibitor incidence was 29.9% (14.9% high-titer [>5 BU]). Sixty-five patients received N8-GP prophylaxis for an average of 2.17 years with a median annualized bleeding rate (interquartile range) of 1.42 (0.76; 3.13) and a 90.5% hemostatic success rate. Temporarily decreased incremental recovery (IR), defined as ≥2 consecutive measurements of IR <0.6 (IU/dL)/(IU/kg) but no inhibitors, was observed in 17 patients within 5 exposure days to N8-GP and had a strong temporal correlation with anti-polyethylene glycol immunoglobulin G antibody titers. IR returned within the expected range with continued N8-GP dosing. During the period of decreased IR, hemostatic response was similar to that of the overall trial population, and no hypersensitivity related to N8-GP or unexpected new adverse events were reported. N8-GP prophylaxis was efficacious for the prevention and treatment of bleeding episodes in PUPs with severe hemophilia A. The inhibitor incidence was 29.9%. All patients with temporarily decreased IR continuing on N8-GP dosing returned within the expected range and had no evident lack of efficacy. This trial was registered at www.clinicaltrials.gov as #NCT02137850.

SUBMITTER: Male C

PROVIDER: S-EPMC9979760 | biostudies-literature | 2023 Feb

REPOSITORIES: biostudies-literature

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The safety and efficacy of N8-GP (turoctocog alfa pegol) in previously untreated pediatric patients with hemophilia A.

Male Christoph C Königs Christoph C Dey Sohan S Matsushita Tadashi T Millner Anders Holm AH Zak Marek M Young Guy G Kenet Gili G

Blood advances 20230201 4

N8-GP (turoctocog alfa pegol) is a recombinant, glycoPEGylated, extended half-life, factor VIII replacement product. Here, we examined the immunogenicity, safety, and efficacy of N8-GP in previously untreated patients (PUPs). pathfinder6 is an ongoing, open-label, phase 3 trial that enrolled PUPs with severe hemophilia A and were aged <6 years. The primary end point was the incidence of factor VIII inhibitors (≥0.6 Bethesda units [BU]). Eighty patients received ≥1 N8-GP dose and were included in ...[more]

PMID: 35858373

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Project description:PurposeTo illustrate the benefits of the extended half-life (EHL) recombinant factor VIII product N8-GP (Esperoct®, turoctocog alfa pegol) by describing individual cases of patients with severe hemophilia A treated with N8-GP in the pathfinder clinical trial program.Patients and methodsThis manuscript presents selected patient cases from the pivotal pathfinder clinical trial program, which included a number of clinical studies in adults (pathfinder 2 and 3) and children (pathfinder 5); overall results published previously. Clinical data and outcomes described in this manuscript are more detailed and derived from several interesting patient cases (five adults from pathfinder 2 and two children from pathfinder 5), who received N8-GP as prophylaxis (PPX) for their severe hemophilia A. Three of the five adults described here also underwent multiple major surgeries (for which they moved from pathfinder 2 into pathfinder 3 and later returned to pathfinder 2). New analyses on pediatric joint health from pathfinder 5 are also summarized here. Outcomes assessed included bleeding complications, improvements in quality of life, intraoperative hemostatic response, blood loss during surgery, number of blood transfusions, and annualized bleeding rates. For the pediatric patients, target joint resolution, adverse events, and annualized joint bleeding rate were also assessed, all by the treating physician.ResultsConsiderable improvements in treatment adherence, bleeding rates, and overall physical activity levels were demonstrated in two adult cases from the pathfinder 2 trial. N8-GP demonstrated good or excellent hemostatic coverage in three adult patients undergoing multiple major surgeries. The benefits of N8-GP for joint health and in support of children and adolescents with evolving active lifestyles were reported for several pediatric cases.ConclusionThese patient cases highlight the benefits of EHL products, such as N8-GP, for patients with severe hemophilia A. They include more challenging scenarios relating to improvements in previously poor adherence to PPX, children with active sporting lifestyles, and patients requiring multiple major surgeries.

Dataset Information

The safety and efficacy of N8-GP (turoctocog alfa pegol) in previously untreated pediatric patients with hemophilia A.

Publications

The safety and efficacy of N8-GP (turoctocog alfa pegol) in previously untreated pediatric patients with hemophilia A.

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