Continuous prophylaxis with recombinant factor IX Fc fusion protein and conventional recombinant factor IX products: comparisons of efficacy and weekly factor consumption

Iorio, Alfonso; Krishnan, Sangeeta; Myrén, Karl-Johan; Lethagen, Stefan; McCormick, Nora; Yermakov, Sander; Karner, Paul

doi:10.1080/13696998.2016.1265973

Cited by 9 publications

(9 citation statements)

References 25 publications

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“…Prophylaxis with long-acting rFIX concentrates has been shown to reduce mean FIX consumption compared with prophylaxis with standard-acting rFIX by almost 50% [84]. Also, in clinical studies with long-acting rFIX concentrates, weekly FIX consumption was considerably lower (~30-50%) than with patients' previous prophylaxis regimens with standard-acting rFIX or pdFIX [34,85].…”

Section: Overcoming Challenges To Prophylaxismentioning

confidence: 98%

The benefits of prophylaxis in patients with hemophilia B

Castaman

2018

Expert Review of Hematology

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The health benefits of prophylactic dosing regimens for clotting factor therapy in patients with hemophilia include reduced joint damage and improved quality of life; as such, prophylaxis is recommended in treatment guidelines. However, many patients with hemophilia B are treated on demand, and prophylaxis has been utilized less frequently than in hemophilia A. Areas covered: This review discusses the opportunities and evidence for prophylaxis in hemophilia B, in the context of treatment guidelines and with regard to factor IX (FIX) replacement therapies, including long-acting recombinant FIX (rFIX). Expert commentary: Long-acting rFIX concentrates may increase uptake of and adherence to prophylaxis regimens through attainment of higher trough levels with longer dosing intervals. In this new era of hemophilia B treatment, physicians may be able to achieve better clinical outcomes for their patients and reconsider treatment goals. Maintaining higher FIX trough levels will undoubtedly have long-term benefits for patients, such as preserving joint function. The long-acting rFIX concentrates support robust prophylaxis regimens and offer physician's flexibility in treating patients to best suit their needs, whether to enable an active lifestyle, to achieve higher trough levels for better bleed protection, or simply to decrease the burden of treatment by reducing injection frequency.

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Section: Overcoming Challenges To Prophylaxismentioning

confidence: 98%

The benefits of prophylaxis in patients with hemophilia B

Castaman

2018

Expert Review of Hematology

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“…85 There is no escape from providing comparative, convincing, robust evidence that the effects of gene therapy are worth its cost. 85 There is no escape from providing comparative, convincing, robust evidence that the effects of gene therapy are worth its cost.…”

Section: Cos T-effec Tivene Ss and He Alth Technology A Ss E Ss Mentmentioning

confidence: 99%

“…70,73,74 For haemophilia, there are established and effective treatments. 85 There are also emerging ones, such as mimetics, recently shown at HTA assessment to be effective, safe and cost-effective, ie, able to reduce societal cost. 86,87 In assessing value, gene therapy will be compared with them.…”

Section: Me a Suring Outcome S In Haemophiliamentioning

confidence: 99%

“…Even with the availability of a concerted core set of outcomes, measuring outcomes is a lengthy and demanding activity, needed both for patients receiving the new treatment and for some receiving the comparator in the real world. 85 There is no escape from providing comparative, convincing, robust evidence that the effects of gene therapy are worth its cost. Patients and their organizations in the community will need to work hard to collect comparative data, and prove to society that the benefits from gene therapy are valuable.…”

Section: Me a Suring Outcome S In Haemophiliamentioning

confidence: 99%

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Past, present and future of haemophilia gene therapy: From vectors and transgenes to known and unknown outcomes

Pierce

Iorio

2018

Haemophilia

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Since the 1960s, the pace of innovation in haemophilia treatment has been fast and furious and occasionally with unintended consequences. As newer technologies are harnessed to better treat, and potentially cure, haemophilias A and B, an understanding of their underlying scientific principles and their benefits and risks are essential for all stakeholders. This review summarizes the starts and stops of introducing FVIII and FIX transgenes clinically, beginning 20 years ago. Lessons from earlier nonclinical and clinical experiments have been utilized to improve vector selection, vector design, promoter/enhancer cis control regions and codon-optimized transgenes to trigger in vivo clinical FVIII and FIX levels in the near-normal to normal ranges. Many known and unknown questions remain, and some, based upon benefit and risk, should be answered during larger phase 3 clinical trials. Prior clinical outcomes in haemophilia trials have not been standardized, making between-trial comparisons difficult. Going forward, haemophilia gene therapy clinical trials should utilize a standard set of core outcomes, to facilitate comparisons to other gene- and protein-based therapies. These outcomes will be more important as the field moves beyond the first-generation gene therapies into more complex vectors that may address the shortcomings of first-generation vectors and offer greater benefits to the patient.

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“…Manco‐Johnson et al 1 were the first to prospectively demonstrate in a randomized study the superiority of prophylaxis over episodic treatment in people with haemophilia. Since and even before this landmark study, the many benefits and values of prophylaxis were demonstrated through multiple prospective and retrospective analyses 2‐11 . In the modern era, prophylaxis is optimized through several approaches, which include varying the dose, varying the dosing frequencies and exploring alternative routes of administration.…”

Section: Introductionmentioning

confidence: 99%

Redefining prophylaxis in the modern era

2020

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Prophylaxis is the globally accepted standard of care for persons with haemophilia and presents many advantages over episodic treatment. The prophylaxis benefits include bleed reduction, reduction in musculoskeletal complications and improvement in the quality of life. The currently evolving novel therapies for the management of haemophilia has ushered a new era characterized by improved prophylaxis targets and outcomes. These redefined targets and outcomes have necessitated the need to also redefine prophylaxis. In this state‐of‐the‐art review, we redefine prophylaxis in the modern era by revisiting its definition, presenting data to support higher trough levels to achieve with prophylaxis and introducing steady‐state haemostasis as a possible new target for prophylaxis.

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Continuous prophylaxis with recombinant factor IX Fc fusion protein and conventional recombinant factor IX products: comparisons of efficacy and weekly factor consumption

Cited by 9 publications

References 25 publications

The benefits of prophylaxis in patients with hemophilia B

The benefits of prophylaxis in patients with hemophilia B

Past, present and future of haemophilia gene therapy: From vectors and transgenes to known and unknown outcomes

Redefining prophylaxis in the modern era

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