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Treatment of congenital bleeding disorders: future trends in management‡
Thrombosis Research 124 Suppl. 2 (2009) S1
Contents lists available at ScienceDirect
Thrombosis Research j o u r n a l h o m e p a g e : w w w. e l s ev i e r. c o m / l o c a t e / t h r o m r e s
Editorial
Treatment of congenital bleeding disorders: future trends in management‡ Donna DiMichelea, *, Pier Mannuccib a Weill b A.
Cornell Medical College, 525 East 68th Street, New York, NY 10065, USA Bianchi Bonomi Hemophilia and Thrombosis Center, Department of Medicine and Medical Specialties, Via Pace, 9, 20122 Milan, Italy
The coagulation pathway is comprised of a complex interaction of many elements, including various coagulation factors, which are essential for stopping the loss of blood at the site of an injury and laying the foundation for injury repair and wound healing. Disruption in any part of this pathway can lead to bleeding that may be serious and life threatening. Defects and/or deficiencies in coagulation factors are common causes of congenital bleeding disorders, such as haemophilia A (factor VIII deficiency), haemophilia B (factor IX deficiency), von Willebrand disease (von Willebrand factor deficiency or dysfunction), and the fibrinogen disorders afibrinogenemia, hypofibrinogenemia and dysfibrinogenemia (fibrinogen deficiency/defect). Our understanding and management of congenital bleeding disorders have improved over the past few decades, resulting in reduced morbidity, improved joint outcomes, enhanced quality of life, and extended life span for patients. Significant advances have also been witnessed in the development of viral reduction and inactivation methods, resulting in improved safety of therapeutics. The introduction of biotechnology into the field of bleeding disorders is potentially offering not only innovative products but also therapeutics with improved functional properties, such as prolonged half-lives and reduced immunogenicity. In this supplement, some of the key issues and future trends in the management of bleeding disorders are addressed.
Steven Pipe provides an informative overview of the recent and ongoing developments in haemophilia care, including the utility of biotechnology and the manufacture of a large pipeline of novel agents. Stefan Schulte discusses the creation of a recombinant factor IX albumin fusion protein with a prolonged half-life, and presents results from his pre-clinical studies. Flora Peyvandi highlights the results of the pharmacokinetic trial of a fibrinogen concentrate in patients with afibrinogenemia. And, lastly, David Bevan compares and contrasts the utility of cryoprecipitate and fibrinogen concentrates for the treatment of congenital fibrinogen disorders. We have really witnessed remarkable progress in the management of bleeding disorders. Although we cannot yet speak of a cure, the scientific and clinical community’s commitment to that goal is leading to some very exciting developments. We feel privileged to be a part of it. Competing interests: DD has no competing interests. PM receives fees as a consultant of Novo Nordisk and Wyeth and as a speaker in educational activities organized by CSL Behring, Baxter, Bayer, Grifols and Kedrion.
* Corresponding author. D. DiMichele: Tel.: +1 212 746 3418; fax: +1 212 746 8986. E-mail address: [email protected] (D. DiMichele).
‡ Proceedings from the CSL Behring-sponsored symposium, held at the XXII Congress of the International Society on Thrombosis and Haemostasis, Boston, USA, 15 July 2009.
0049-3848 /$ – see front matter © 2009 Elsevier Ltd. All rights reserved.
Contents lists available at ScienceDirect
Thrombosis Research j o u r n a l h o m e p a g e : w w w. e l s ev i e r. c o m / l o c a t e / t h r o m r e s
Editorial
Treatment of congenital bleeding disorders: future trends in management‡ Donna DiMichelea, *, Pier Mannuccib a Weill b A.
Cornell Medical College, 525 East 68th Street, New York, NY 10065, USA Bianchi Bonomi Hemophilia and Thrombosis Center, Department of Medicine and Medical Specialties, Via Pace, 9, 20122 Milan, Italy
The coagulation pathway is comprised of a complex interaction of many elements, including various coagulation factors, which are essential for stopping the loss of blood at the site of an injury and laying the foundation for injury repair and wound healing. Disruption in any part of this pathway can lead to bleeding that may be serious and life threatening. Defects and/or deficiencies in coagulation factors are common causes of congenital bleeding disorders, such as haemophilia A (factor VIII deficiency), haemophilia B (factor IX deficiency), von Willebrand disease (von Willebrand factor deficiency or dysfunction), and the fibrinogen disorders afibrinogenemia, hypofibrinogenemia and dysfibrinogenemia (fibrinogen deficiency/defect). Our understanding and management of congenital bleeding disorders have improved over the past few decades, resulting in reduced morbidity, improved joint outcomes, enhanced quality of life, and extended life span for patients. Significant advances have also been witnessed in the development of viral reduction and inactivation methods, resulting in improved safety of therapeutics. The introduction of biotechnology into the field of bleeding disorders is potentially offering not only innovative products but also therapeutics with improved functional properties, such as prolonged half-lives and reduced immunogenicity. In this supplement, some of the key issues and future trends in the management of bleeding disorders are addressed.
Steven Pipe provides an informative overview of the recent and ongoing developments in haemophilia care, including the utility of biotechnology and the manufacture of a large pipeline of novel agents. Stefan Schulte discusses the creation of a recombinant factor IX albumin fusion protein with a prolonged half-life, and presents results from his pre-clinical studies. Flora Peyvandi highlights the results of the pharmacokinetic trial of a fibrinogen concentrate in patients with afibrinogenemia. And, lastly, David Bevan compares and contrasts the utility of cryoprecipitate and fibrinogen concentrates for the treatment of congenital fibrinogen disorders. We have really witnessed remarkable progress in the management of bleeding disorders. Although we cannot yet speak of a cure, the scientific and clinical community’s commitment to that goal is leading to some very exciting developments. We feel privileged to be a part of it. Competing interests: DD has no competing interests. PM receives fees as a consultant of Novo Nordisk and Wyeth and as a speaker in educational activities organized by CSL Behring, Baxter, Bayer, Grifols and Kedrion.
* Corresponding author. D. DiMichele: Tel.: +1 212 746 3418; fax: +1 212 746 8986. E-mail address: [email protected] (D. DiMichele).
‡ Proceedings from the CSL Behring-sponsored symposium, held at the XXII Congress of the International Society on Thrombosis and Haemostasis, Boston, USA, 15 July 2009.
0049-3848 /$ – see front matter © 2009 Elsevier Ltd. All rights reserved.