A comparison of methods for prediction of pharmacokinetics across factor concentrate switching in hemophilia patients

Yu, Jacky K.; Iorio, Alfonso; Chelle, Pierre; Edginton, Andrea N.

doi:10.1016/j.thromres.2019.10.023

Cited by 5 publications

(6 citation statements)

References 26 publications

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“…This method uses information from the time of receiving octocog alfa to predict the outcome on damoctocog alfa pegol. This method is described as Method 2 in Yu et al 19 …”

Section: Methodsmentioning

confidence: 99%

“…This method uses information from the time of receiving octocog alfa to predict the outcome on damoctocog alfa pegol. This method is described as Method 2 in Yu et al 19 Observed pharmacokinetic outcomes, as estimated from Bayesian modelling of the measured post-infusion levels, of damoctocog alfa pegol for each patient were compared to the predicted values derived from the predictive model based on measured plasma levels of octocog alfa before the switch. The coefficient of determination was used to…”

Section: Modelling To Predict Product Switch From Octocog Alfa To Dam...mentioning

confidence: 99%

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Canadian clinical experience on switching from standard half‐life recombinant factor VIII (rFVIII), octocog alfa, to extended half‐life rFVIII, damoctocog alfa pegol, in persons with haemophilia A ≥ 12 years followed in a Comprehensive Hemophilia Care Program in Canada

Matino,

Germini,

Chan

et al. 2024

Haemophilia

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IntroductionDamoctocog alfa pegol (BAY 94–9027, Jivi®) is an extended half‐life recombinant factor (F)VIII replacement, indicated for the treatment of haemophilia A in patients aged ≥12 years. Following introduction of damoctocog alfa pegol in Canada in 2020, there have been no reports on routine clinical effectiveness and satisfaction, when switching from a previous FVIII product in Canada.AimTo report changes in pharmacokinetics, effectiveness, utilization and patient satisfaction when switching to damoctocog alfa pegol prophylaxis from previous standard half‐life octocog alfa (BAY 81–8973, Kovaltry®) treatment.MethodsA single‐centre, intra‐patient comparison of pharmacokinetics and clinical outcomes was performed. Blood samples drawn once pre‐dose and ≥2 times post‐dose were measured by a one‐stage assay to assess pharmacokinetic parameters including area under the curve (AUC, primary endpoint). Patient‐reported outcomes data were collected using the Patient‐Reported Outcomes, Burdens and Experiences questionnaire (PROBE). Clinical outcomes included annualized bleeding rate (ABR) and factor utilization.ResultsDose‐normalized AUC was significantly increased after switch to damoctocog alfa pegol from octocog alfa. Median (quartile [Q]1; Q3) annualized bleeding rates were 0.67 (0.00; 1.33) with damoctocog alfa pegol and 1.33 (0.00; 2.67) with octocog alfa. Half of the patients receiving damoctocog alfa pegol prophylaxis experienced zero bleeds (n = 9, 50.0%) versus 38.9% (n = 7) of patients treated with octocog alfa. Patients’ good quality of life was maintained.ConclusionThis study provides routine clinical evidence supporting the benefits of switching from octocog alfa to damoctocog alfa pegol for patients with severe haemophilia A.

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“…This method uses information from the time of receiving octocog alfa to predict the outcome on damoctocog alfa pegol. This method is described as Method 2 in Yu et al 19 …”

Section: Methodsmentioning

confidence: 99%

Section: Modelling To Predict Product Switch From Octocog Alfa To Dam...mentioning

confidence: 99%

Canadian clinical experience on switching from standard half‐life recombinant factor VIII (rFVIII), octocog alfa, to extended half‐life rFVIII, damoctocog alfa pegol, in persons with haemophilia A ≥ 12 years followed in a Comprehensive Hemophilia Care Program in Canada

Matino,

Germini,

Chan

et al. 2024

Haemophilia

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“…Digitalization of health records was a game changer in the coagulation department, allowing us to follow drug pharmacokinetics via mobile applications (i.e., WAPPS-Hemo and myWAPPS), store pictures in the electronic systems, retrieve old data, and most importantly do more clinical and translational research. 12 13 The synergy between the clinic and laboratory continually benefits from technological advancements. Automation, robotics, and high-throughput techniques streamline laboratory workflows, reducing turnaround times, and enhancing precision in test results.…”

Section: From the Clinics To The Laboratory: How Can Laboratory Best ...mentioning

confidence: 99%

“…Furthermore, ongoing patient monitoring involves regular laboratory assessments to track treatment efficacy, adjust medication dosages, and anticipate potential complications. 13 For instance, monitoring factor levels in hemophilia patients helps in adjusting replacement therapy doses, ensuring cost-effective care. Anticoagulation monitoring of vitamin K antagonists, development of point-of-care services, as well as assays for direct anticoagulants for management in acute settings are also novel major functions of the coagulation laboratories.…”

Section: From the Clinics To The Laboratory: How Can Laboratory Best ...mentioning

confidence: 99%

Development of a Coagulation Disorders Unit

Szanto,

Helin,

Joutsi-Korhonen

et al. 2024

Semin Thromb Hemost

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Our Coagulation Disorders Unit in Helsinki, Finland, provides 24/7 services for local and national hospitals and colleagues upon requests regarding bleeding and thrombosis diagnostics and management, including follow-up. The unit has a tight connection between the clinic and laboratory, and its maintenance and sharing knowledge and observations have been priorities, already for over 20 years and will continue to be of major importance. The consultation service is provided by phone during daytime and on-call hours, and in written form sent electronically to the consulting stakeholders. Thrombosis and hemostasis-targeted outpatient clinics are also available for the patients referred to the center. Writing local guidance and official guidelines, Nordic, European and international collaboration, and educational activities including social communication are critical elements for the Coagulation Disorders Unit. Alertness to acute coagulation abnormalities, such as occurred during COVID-19 and vaccine-induced thrombosis and thrombocytopenia, and development of strategies to manage cross-disciplinary problems are topics which call upon broad networking. The Nordic community has an ongoing historical meeting, which has been circulating among coagulation centers for the past 56 years. At the European level, the European Association of Haemophilia and Allied Disorders focuses on bleeding disorders and their management, including safety surveillance. The International Society of Thrombosis and Haemostasis offers excellent basic and clinical benchmarks for any Coagulation Disorders Unit. We hope that the description of the development and implementation of our Coagulation Disorders Unit in Helsinki achieves international interest and broadens international collaboration. Finally, we congratulate STH on its great contributions around the globe and for providing a vivid forum to foster the discipline of thrombosis and hemostasis.

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“…51 These analyses provide objective and accurate parameters, and last over time, with a close correspondence between observed and predicted results. 52 The design of a PK-guided individualized prophylaxis protocol has also been shown to improve adherence, 53 allows specialists to objectively decide when the switch to other products is a suitable option, 54 and saves factor consumption-related costs. 55…”

Section: Pharmacokinetics/medication Propertiesmentioning

confidence: 99%

The Limitations and Unmet Needs of the Five Cornerstones to Guarantee Lifelong Optimization of Prophylaxis in Hemophilia Patients

Núñez

Álvarez‐Román²,

Bonanad

et al. 2022

TH Open

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Prophylaxis to prevent bleeding is highly recommended for hemophilia patients. The development of new drugs and tools for modeling personalized prophylaxis provides the means for people with hemophilia to lead active lives with a quality of life comparable to that of nonhemophilic individuals. The choice of regimens must be made on a highly individual basis. Unfortunately, reference guides neither always concur in their recommendations nor provide directions to cover all possible scenarios. In this review, a group of experts identify the significant limitations and unmet needs of prophylaxis, taking advantage of their clinical experience in the disease, and supported by a rigorous literature update. To perform a more systematic and comprehensive search for gaps, the main cornerstones that influence decisions regarding prophylactic patterns were first identified.Bleeding phenotype, joint status, physical activity, pharmacokinetics/medication properties, and adherence to treatment were considered as the primary mainstays that should allow physicians guiding prophylaxis to secure the best outcomes. Several challenges identified within each of these topics require urgent attention and agreement. The scores to assess severity of bleeding are not reliable, and lead to no consensus definition of severe bleeding phenotype. The joint status is to be redefined in light of new, more efficient treatments with an agreement to establish one scale as the unique reference for joint health. Further discussion is needed to establish the appropriateness of high-intensity physical activities according to patient profiles, especially because sustaining trough factor levels within the safe range is not always warranted for long periods. Importantly, many physicians do not benefit from the advantages provided by the programs based on population pharmacokinetic models to guide individualized prophylaxis through more efficient and cost-saving strategies. Finally, ensuring correct adherence to long-term treatments may be time-consuming for practitioners, who often have to encourage patients and review complex questionnaires.In summary, we identify five cornerstones that influence prophylaxis and discuss the main conflicting concerns that challenge the proper long-term management of hemophilia. A consensus exercise is warranted to provide reliable guidelines and maximize benefit from recently developed tools that should notably improve patients' quality of life.

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A comparison of methods for prediction of pharmacokinetics across factor concentrate switching in hemophilia patients

Cited by 5 publications

References 26 publications

Canadian clinical experience on switching from standard half‐life recombinant factor VIII (rFVIII), octocog alfa, to extended half‐life rFVIII, damoctocog alfa pegol, in persons with haemophilia A ≥ 12 years followed in a Comprehensive Hemophilia Care Program in Canada

Canadian clinical experience on switching from standard half‐life recombinant factor VIII (rFVIII), octocog alfa, to extended half‐life rFVIII, damoctocog alfa pegol, in persons with haemophilia A ≥ 12 years followed in a Comprehensive Hemophilia Care Program in Canada

Development of a Coagulation Disorders Unit

The Limitations and Unmet Needs of the Five Cornerstones to Guarantee Lifelong Optimization of Prophylaxis in Hemophilia Patients

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