Clotting factor concentrates for preventing bleeding and bleeding-related complications in previously treated individuals with haemophilia A or B

Olasupo, Omotola; Lowe, Megan; Krishan, Ashma; Collins, Peter William; Iorio, Alfonso; Matino, Davide

doi:10.1002/14651858.cd014201

Cited by 9 publications

(7 citation statements)

References 152 publications

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“…Extensive studies have demonstrated the benefits of regular long‐term factor replacement therapy in protecting from hemarthroses and joint damage 1,2 . In haemophilia A, standard FVIII prophylaxis is administered intravenously every other day or at least three times weekly 3,4 . The need for repetitive venous access is one of the main burdens of prophylaxis 5 .…”

Section: Introductionmentioning

confidence: 99%

“…1,2 In haemophilia A, standard FVIII prophylaxis is administered intravenously every other day or at least three times weekly. 3,4 The need for repetitive venous access is one of the main burdens of prophylaxis. 5 Frequent dosing aims at maintaining FVIII activity always above a threshold value (at least 1%-5%), though the minimal trough level which will prevent joint bleeds varies between patients.…”

Section: Introductionmentioning

confidence: 99%

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Switching from standard to extended half‐life FVIII prophylaxis in haemophilia A: Comparison of factor product use, bleed rates and pharmacokinetics

et al. 2022

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Introduction Majority of haemophilia A patients in our comprehensive care centre have switched from standard half‐life (SHL) to extended half‐life (EHL) FVIII products in a short time. Aim We compared the clinical and laboratory outcomes between SHL and EHL FVIII prophylaxis in product switchers. Methods This is a retrospective inception cohort of all adult haemophilia A patients switched to EHL (rFVIIIFc or rFVIII‐PEG) prophylaxis in our centre. Dosing, product utilization, annualized bleed rates (ABR), treatment regimen and pharmacokinetics by Web Accessible Population Pharmacokinetic Service (WAPPS)‐Hemo were compared between SHL and EHL. Results We included 38 patients, whose median age was 38 years (range 17–75). Median FVIII dose was 23 IU/kg for SHL versus 25 IU/kg for EHL. After switching, weekly infusions decreased by 29% from median 2.8 (every 2.5 days) to 2.0 (every 3.5 days) (P = <.001) and factor consumption for prophylaxis by 17% from 60 to 50 IU/kg/week (P = <.001). Weekly infusions decreased in 71% and FVIII utilization in 55% of patients. ABR remained low (1.0 for SHL and .5 for EHL, respectively). In pharmacokinetics, the half‐life of FVIII increased from median 13 to 21 h after switching. Times above .01 and .03 IU/ml improved from 85 to 131 h and from 65 to 106 h. Half‐lives of the SHL products and von Willebrand factor levels predicted half‐lives with the EHL products. Conclusions Our cohort study confirms the successful experience of switching to EHL FVIII products, with decreased infusion frequency, factor consumption and excellent clinical efficacy.

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Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Switching from standard to extended half‐life FVIII prophylaxis in haemophilia A: Comparison of factor product use, bleed rates and pharmacokinetics

et al. 2022

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“…1 When optimally administered, prophylaxis can decrease the frequency of joint haemorrhages, prevent resultant joint damage and reduce the development of chronic haemophilic arthropathy. [2][3][4] Standard half-life FVIII products, which include plasma-derived FVIII and recombinant FVIII (rFVIII) products, have relatively short half-lives, and so require more frequent intravenous infusions (two to three times per week or every other day), which represents a significant treatment burden for patients and caregivers. 5 The need for frequent intravenous infusions can lead to suboptimal adherence, 6,7 potentially leading to an increased time with low FVIII levels and higher occurrence of breakthrough bleeding and haemarthroses.…”

Section: Introductionmentioning

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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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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“…1 The standard of care for severe haemophilia is regular prophylaxis, which substantially reduces bleeds compared with on-demand treatment. 2 Prophylaxis aims to protect against spontaneous bleeding episodes and prolonged bleeds after trauma, thereby preventing or reducing joint disease, maintaining musculoskeletal health, and improving life expectancy and quality of life. 1 Historically, prophylaxis in severe haemophilia with replacement FVIII therapy has targeted trough FVIII levels in the moderate haemophilia range, based on the observation that people with moderate haemophilia have a lower risk of bleeds.…”

Section: Introductionmentioning

confidence: 99%

“…The severe form is characterised by spontaneous bleeds, often into joints and muscles 1 . The standard of care for severe haemophilia is regular prophylaxis, which substantially reduces bleeds compared with on‐demand treatment 2 . Prophylaxis aims to protect against spontaneous bleeding episodes and prolonged bleeds after trauma, thereby preventing or reducing joint disease, maintaining musculoskeletal health, and improving life expectancy and quality of life 1 .…”

Section: Introductionmentioning

confidence: 99%

Targeting higher factor VIII levels for prophylaxis in haemophilia A: a narrative review

Malec,

Matino

2023

Haemophilia

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IntroductionThe standard of care in severe haemophilia A is prophylaxis, which has historically aimed for a factor VIII (FVIII) trough level of ≥1%. However, despite prophylactic treatment, people with haemophilia remain at risk of bleeds that have physical and quality of life implications, and that impact everyday life.AimThe aim of this review was to evaluate evidence supporting the relationship between targeting higher FVIII activity levels with prophylaxis and improved outcomes in people with haemophilia A.MethodsWe conducted a narrative review that defined the unmet needs and treatment goals in people with haemophilia A, evaluated evidence to support targeting higher FVIII activity levels, and highlighted therapies that may support higher and sustained FVIII activity levels and improved outcomes for people with haemophilia A.ResultsDespite recent advances in treatment, unmet needs remain, and people with haemophilia continue to experience joint and functional impairment, acute and chronic pain, and poor mental health. All these negatively impact their health‐related quality of life. Evidence suggests that FVIII activity levels of up to 50% may be needed to achieve a near‐zero joint bleed rate. However, achieving high FVIII activity levels with current standard and extended half‐life (EHL) FVIII replacement therapies is associated with a high treatment burden. Innovative treatment options may provide high sustained FVIII activity levels and improved patient outcomes.ConclusionEvidence suggests that FVIII activity levels in people with haemophilia A should be sustained at higher levels to improve joint and patient outcomes and enable progression towards health equity.

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Clotting factor concentrates for preventing bleeding and bleeding-related complications in previously treated individuals with haemophilia A or B

Cited by 9 publications

References 152 publications

Switching from standard to extended half‐life FVIII prophylaxis in haemophilia A: Comparison of factor product use, bleed rates and pharmacokinetics

Switching from standard to extended half‐life FVIII prophylaxis in haemophilia A: Comparison of factor product use, bleed rates and pharmacokinetics

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

Targeting higher factor VIII levels for prophylaxis in haemophilia A: a narrative review

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