The case for wider use of recombinant factor VIII concentrates

Hermans, Cédric; Brackmann, Hans‐Hermann; Schinco, Piercarla; Auerswald, Günter

doi:10.1016/j.critrevonc.2011.08.001

Cited by 11 publications

(6 citation statements)

References 54 publications

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“… 36 Still, concerns remain regarding the transmission of other pathogens, including nonenveloped viruses such as the parvoviruses and hepatitis A, or prions (variant Creutzfeldt-Jakob disease). 35 , 37 , 38 …”

Section: Standard Hemophilia Managementmentioning

confidence: 99%

Consensus Review of the Treatment of Cardiovascular Disease in People With Hemophilia A and B

Ferraris

Boral

Cohen

et al. 2015

Cardiology in Review

104

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With advances in care, increasing numbers of people with hemophilia (PWH) achieve near-normal life expectancies and present with typical age-related cardiovascular conditions. Evidence-based guidelines for medical or surgical management of cardiovascular conditions in individuals with hemophilia are limited. Published recommendations exist for the management of some common cardiovascular conditions (eg, ischemic heart disease, atrial fibrillation), but identifying optimal strategies for anticoagulant or antithrombotic therapy constitutes the primary challenge of managing nonoperative cardiovascular disease (CVD) in PWH. In general, as long as factor concentrates or other hemostatic therapies maintain adequate hemostasis, the recommended medical and surgical management of CVD in PWH parallels that in individuals without hemophilia. The presence of factor inhibitors complicates hemophilia management. Published outcomes of CVD treatment in PWH are similar to those in the general population. Specific knowledge about factor replacement, factor inhibitors, and disease-specific treatment distinguishes the cardiovascular care of PWH from similar care of individuals without this rare bleeding disorder. Furthermore, a multidisciplinary approach incorporating a hematologist with an onsite coagulation laboratory, ideally associated with a hemophilia treatment center, is integral to the management of CVD in PWH.

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Section: Standard Hemophilia Managementmentioning

confidence: 99%

Consensus Review of the Treatment of Cardiovascular Disease in People With Hemophilia A and B

Ferraris

Boral

Cohen

et al. 2015

Cardiology in Review

104

View full text Add to dashboard Cite

show abstract

“…This is important because, from 1990 to 2006, a three-fold increase in the worldwide use of FVIII was seen (8), and dynamics remain unchanged; e. g. there was a 12% increase in FVIII use from 2011 to 2012, based on data from 50 countries (9). The most important way to meet the increased worldwide FVIII requirement is the use of rFVIII products, as it is increasingly difficult to improve the yield of pd-FVIII products (10). Moreover, the production of pd-FVIII products is challenged by the potential risk of transmission of bloodborne viral or non-viral pathogens, requiring careful selection and screening of plasma donors and constantly evolving methods of pathogen screening and removal (11).…”

Section: Treatment Options For Patients With Congenital Haemophilia Amentioning

confidence: 99%

Turoctocog alfa (recombinant factor VIII)

Tiede

Klamroth²,

Oldenburg³

2015

Hamostaseologie

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SummaryTuroctocog alfa (NovoEight ® ) is a new recombinant factor VIII (rFVIII) with a truncated B domain and a high degree of tyrosine sulphation, similar to plasma-derived FVIII products. The manufacturing process includes double nanofiltration with a 20-nm pore size and immunoaffinity chromatography with monoclonal F25 anti-FVIII antibodies. Treatment with turoctocog alfa can be monitored with both one-stage and chromogenic substrate assays without a product-specific laboratory standard. In total, 213 previouslytreated patients with severe haemophilia A participated in the pivotal part of the clinical trial programme guardian TM . The median annualised bleeding rate during turoctocog alfa prophylaxis was 3.7 and 3.0 in adolescents/adults and children, respectively, with marked differences between participating countries. The success rate for the treatment of breakthrough bleeds was 85% (adults/ adolescents) and 94% (children

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“…The intermediated-purity concentrates that are produced of large pools or huge numbers of donors' plasma have 2-5 units of factor VIII per milligram protein. There are noticeable amount of von Willebrand factor (vWF) in them [19]. The high purity concentrates are manufactured using monoclonal antibodies to factor VIII or vWF.…”

Section: Plasma Derived Factor VIII Concentrates (Pdfviiic)mentioning

confidence: 99%

The Current Role of Bone Marrow Transplantation in the Treatment of Hemophilia A

Mansouritorghabeh

Ghasemi²

2014

J Hematol Thrombo Dis

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Bone marrow transplantation (BMT) is nowadays used in various hematological disorders including leukemias. Hemophilia A is sex linked bleeding disorder in which there are various genetic abnormalities in factor VIII gene. Among various hematological disorders, bleeding disorders mainly hemophilia in now widely treated using plasma derived and recombinant factor VIII concentrates. Day to day transfusion of coagulation factor VIII in current lifelong disorder is a burden for the patients and decreases their quality of life. Although infection of coagulation products with blood-borne viruses that was major concern in treatment of hemophilia has been eliminated nearly complete at the present time, but appearance of factor VIII inhibitor is huge burden in treatment of hemophilia for both clinicians and patients. In this day and age BMT has gathered new insight and acceptable treatment for many hematological and non-hematological disorders. If BMT will be successful in treatment of hemophilia, it will improve quality of life (by nearly permanent treatment without necessary of daily treatment) and decrease mortality rate (by providing continuous coagulation factor level in blood system). The aim of current review was to determine current role of BMT in treatment of hemophilia and to evaluate current usage of BMT for treatment of hemophilia. The review of literatures showed that although BMT has been used in a rare cases with hemophilia (mainly hemophilia with another blood disorder), it does not use commonly in treatment of patients with hemophilia A at present time. Literature review showed that scientists now focused on bone marrow transplantation in animal model of hemophilia.

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The case for wider use of recombinant factor VIII concentrates

Cited by 11 publications

References 54 publications

Consensus Review of the Treatment of Cardiovascular Disease in People With Hemophilia A and B

Consensus Review of the Treatment of Cardiovascular Disease in People With Hemophilia A and B

Turoctocog alfa (recombinant factor VIII)

The Current Role of Bone Marrow Transplantation in the Treatment of Hemophilia A

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