Haemophilic patients with inhibitors to factor VIII or IX: variables affecting treatment response

Penner, J. A.

doi:10.1111/j.1365-2516.2001.00473.x

Cited by 7 publications

(7 citation statements)

References 33 publications

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“…Genetic risk factors including race, ethnicity, FVIII mutation type and severity of disease, in addition to therapy‐related factors including quantity and time of FVIII product exposure, can all influence inhibitor development (89, 103). In addition to the type of ITI therapy, the following variables seem to be significant patient predictors of unsuccessful ITI (81, 86, 91, 94, 104):…”

Section: Treatmentmentioning

confidence: 99%

A systematic overview of the first pasteurised VWF/FVIII medicinal product, Haemate^®P/ Humate^®‐P: History and clinical performance

Berntorp

Archey

Auerswald

et al. 2008

European J of Haematology

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Patients with von Willebrand disease (VWD) and haemophilia A (HA) lack, to varying degrees, the von Willebrand factor (VWF) and coagulation factor VIII (FVIII) that are critical for normal haemostasis. These conditions in turn make patients prone to uncontrolled bleeding. Historically, patients with severe forms of VWD or HA were crippled before adulthood and their life expectancy was significantly reduced. Over the past decades, specific coagulation factor replacement therapies including Haemate Ò P, have been developed to help patients achieve and maintain normal haemostasis. Haemate Ò P is a human, plasma-derived VWF ⁄ FVIII medicinal product, which was first licensed in Germany in 1981 for the treatment of HA-associated bleeding. It has since then come to be accepted as the gold standard for both the treatment and prophylaxis of bleeding in VWD, especially in cases where desmopressin [1-deamino-8-D-arginine vasopressin (DDAVP)] has been ineffective. Haemate Ò P was the first effectively virus-inactivated (pasteurisation: 60°C for 10 h in aqueous solution) FVIII product, whereby the risk of potentially threatening infective complications of plasma-derived products was reduced. Haemate Ò P was also shown to have a VWF multimer profile remarkably close to that of normal plasma. This bibliographic review presents previously unpublished clinical data of Haemate Ò P, based upon internal clinical study reports of the proprietor, CSL Behring, in addition to data already presented in other publications. The data demonstrate a predictable and well-characterised pharmacokinetic profile, and a proven record of short-and long-term safety, while effectively correcting the haemostatic defects in VWD and HA. Recently available data have also shown Haemate Ò P to be of haemostatic value in exceptional clinical circumstances including surgical interventions. By virtue of its plasma-derived combination of VWF and FVIII, in addition to its high VWF:FVIII content ratio (2.4:1), Haemate Ò P is also associated with successful immune tolerance induction in those patients developing inhibitor antibodies. Although the theoretical risk of thromboembolic complications does exist while receiving Haemate Ò P, as it does with any FVIII replacement therapy, the incidence of such complications has remained notably low. Given the robust data that have accumulated for the use of Haemate Ò P, dosing recommendations are also described in this review; the recommendations are tailored to patient-specific contexts including baseline VWF and FVIII levels in plasma and the type of surgical intervention being undertaken. A wide variety of studies have also provided data on paediatric and geriatric populations, all of which have suggested that Haemate Ò P can be safely and effectively used in a wide variety of clinical circumstances. Effective haemostasis requires complex bodily processes such as vasoconstriction and blood clotting so as to ensure the integrity of the blood vessel system and to prevent excessive blood loss following injury. B...

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Section: Treatmentmentioning

confidence: 99%

A systematic overview of the first pasteurised VWF/FVIII medicinal product, Haemate^®P/ Humate^®‐P: History and clinical performance

Berntorp

Archey

Auerswald

et al. 2008

European J of Haematology

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“…Secondly, among 33% of severely and moderately affected haemophilia A individuals, exogenous clotting factor replacement therapy stimulates an alloantibody (inhibitor) response to Factor VIII [18]. Over half of these neutralizing antibodies are high titre and exhibit anamnesis, precluding ongoing specific factor replacement [19,20]. Such individuals are susceptible to increased morbidity and mortality from their haemophilia [21].…”

Section: Making the Choice For Gene Therapymentioning

confidence: 99%

Gene therapy ethics and haemophilia: an inevitable therapeutic future?

DiMichele¹,

Miller

Fins

2003

Haemophilia

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Haemophilia was recognized early on as an ideal candidate for a gene transfer approach to therapy. In the past decade, gene transfer experimentation in the haemophilias has indeed played an integral role in furthering the science in the global field of gene therapy. However, these expectations have placed haemophilia gene transfer researchers under pressure to succeed in a scientific domain in which successes are infrequent and progress is necessarily slow. These same expectations have also fueled the perception of gene therapy as the inevitable therapeutic goal for the youngest children with haemophilia. In this paper, we will discuss the ethical implications of this perception in light of anticipated benefits, acceptable risk, perceived consumer need and the unknown cost of this intervention. A framework for the future study and therapeutic implementation of gene transfer technology in this specific population is proposed. Public debate on this issue that includes the voices of the intended beneficiaries, especially the parents of the youngest children with haemophilia and the children themselves, is encouraged.

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“…Maintaining effective treatment for haemophilia A patients with inhibitor responses remains a challenge, despite the relatively recent development of alternative methods of treatment (Hacker et al, 2001; Kulkarni et al, 2001; Penner, 2001; Rossi, 2001). Low‐titre inhibitors [measured as titres of ≤5 Bethesda units (BU)/ml] pose a relatively tractable problem, with therapy consisting of either hormone‐induced secretion of factor VIII by DDAVP or by simply overcoming the inhibitor response by overwhelming the antibodies with larger amounts of therapeutic factor VIII during infusions.…”

Section: Current Treatments For Antibody Inhibitorsmentioning

confidence: 99%

Optimization of factor VIII replacement therapy: can structural studies help in evading antibody inhibitors?

Spiegel

Stoddard

2002

Br J Haematol

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Haemophilic patients with inhibitors to factor VIII or IX: variables affecting treatment response

Cited by 7 publications

References 33 publications

A systematic overview of the first pasteurised VWF/FVIII medicinal product, Haemate^®P/ Humate^®‐P: History and clinical performance

A systematic overview of the first pasteurised VWF/FVIII medicinal product, Haemate^®P/ Humate^®‐P: History and clinical performance

Gene therapy ethics and haemophilia: an inevitable therapeutic future?

Optimization of factor VIII replacement therapy: can structural studies help in evading antibody inhibitors?

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Haemophilic patients with inhibitors to factor VIII or IX: variables affecting treatment response

Cited by 7 publications

References 33 publications

A systematic overview of the first pasteurised VWF/FVIII medicinal product, Haemate®P/ Humate®‐P: History and clinical performance

A systematic overview of the first pasteurised VWF/FVIII medicinal product, Haemate®P/ Humate®‐P: History and clinical performance

Gene therapy ethics and haemophilia: an inevitable therapeutic future?

Optimization of factor VIII replacement therapy: can structural studies help in evading antibody inhibitors?

Contact Info

Product

Resources

About

A systematic overview of the first pasteurised VWF/FVIII medicinal product, Haemate^®P/ Humate^®‐P: History and clinical performance

A systematic overview of the first pasteurised VWF/FVIII medicinal product, Haemate^®P/ Humate^®‐P: History and clinical performance