Dataset Information

A field study evaluating the activity of N8-GP in spiked plasma samples at clinical haemostasis laboratories.

ABSTRACT:

Aim

N8-GP (turoctocog alfa pegol) is a glycoPEGylated, extended half-life human recombinant factor VIII (FVIII) shown to be an efficacious treatment for patients with haemophilia A. Accurate monitoring of replacement therapy helps ensure proper dosing, leading to better patient care. The objective of this field study was to evaluate the accuracy and intra- and inter-laboratory variabilities of N8-GP and rAHF (Advate^® ) FVIII activity (FVIII:C) measurements in clinical laboratories using their routine methods and reagents.

Methods

Laboratories measured plasma samples spiked with 0.03, 0.2, 0.6 and 0.9 IU/mL N8-GP or rAHF. Samples were blinded, and laboratories were instructed to perform evaluations using their routine FVIII activity assays and calibrators.

Results

Of the 67 participating laboratories from 25 countries, 60 used a one-stage assay, 36 used a chromogenic assay, and 29 used both one-stage and chromogenic assays. Participating laboratories used nine different activated partial thromboplastin time (aPTT) reagents, the most common being SynthASil^® and Actin^® FS. Most aPTT reagents recovered N8-GP close to target. Three silica-based aPTT reagents (APTT-SP, TriniCLOT™ and STA^® PTT-Automate) underestimated N8-GP, recovering 40%-83% of target concentration. For chromogenic assays, N8-GP and rAHF recoveries were comparable at all concentrations, with overall mean recoveries for both products close to 130%. Assay variability was similar for both assay types and both products; inter-laboratory variability was greater than intra-laboratory variability and highest at 0.03 IU/mL.

Conclusions

Most clinical laboratories accurately measured N8-GP and rAHF when using their in-house one-stage or chromogenic FVIII:C assays. However, three silica-based aPTT reagents underestimated N8-GP recovery.

SUBMITTER: Tiefenbacher S

PROVIDER: S-EPMC6852407 | biostudies-literature | 2019 Sep

REPOSITORIES: biostudies-literature

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Publications

A field study evaluating the activity of N8-GP in spiked plasma samples at clinical haemostasis laboratories.

Tiefenbacher Stefan S Clausen Wan Hui Ong WHO Hansen Martin M Lützhøft Rasmus R Ezban Mirella M

Haemophilia : the official journal of the World Federation of Hemophilia 20190711 5

<h4>Aim</h4>N8-GP (turoctocog alfa pegol) is a glycoPEGylated, extended half-life human recombinant factor VIII (FVIII) shown to be an efficacious treatment for patients with haemophilia A. Accurate monitoring of replacement therapy helps ensure proper dosing, leading to better patient care. The objective of this field study was to evaluate the accuracy and intra- and inter-laboratory variabilities of N8-GP and rAHF (Advate<sup>®</sup> ) FVIII activity (FVIII:C) measurements in clinical laborato ...[more]

PMID: 31294905

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Project description:BACKGROUND:Switzerland lacks future general practitioners (GPs). Residents who wished to specialize as general practitioners were formerly trained solely in hospital settings. To better prepare and also attract more young doctors to become GPs, the canton of Bern (equivalent to a state) has implemented a partly state-funded vocational training program in GP practices. Our study examines the efficacy of this 10-year program, identifies factors that positively influence residents in their decision to become a GP and the distribution of new GPs in the canton of Bern, who had taken part in the traineeship. METHODS:This cross-sectional survey among all residents, who participated in a traineeship in general practice from 2008 to 2017 in the canton of Bern asked if residents had taken a subsequent career choice as a GP and if so in which region. Residents scored the importance of their traineeship and their mentor's influence on becoming a GP. By using zip codes of work area of respondents already working as GPs and matching it with population census data, we could obtain the distribution of GPs on a per capita basis. RESULTS:Out of 165 residents who participated in a traineeship, 151 (92%) completed our survey. 81% had chosen a career as a GP or were on track to become a GP. Almost half of the participants became GPs in the offices of their mentors or in the area. Our respondents emphasized the importance of their mentors' influence as well as the training program in their decision-making to become a GP. Most mentioned benefits of being a GP were broad field of medical care (37%) and a fulfilling doctor-patient relationship (34%). We could show an increase in GP practices in the canton of Bern, not only in urban but also accordingly in rural areas. CONCLUSIONS:Most residents continued subsequent careers as general practitioners after having completed a GP traineeship, with almost half of them in the region of their training. A vocational training program helped motivating young doctors to become GPs and underserved regions of the canton of Bern to gain new GPs.

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

A field study evaluating the activity of N8-GP in spiked plasma samples at clinical haemostasis laboratories.

Aim

Methods

Results

Conclusions

Publications

A field study evaluating the activity of N8-GP in spiked plasma samples at clinical haemostasis laboratories.

Similar Datasets

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