Naveen Manchanda, MD
2010-12-04
Remarkable progress has been made in the field of hemophilia in the last half century. However, it was only in the ’80s and ’90s that we saw the cruel consequences of administering contaminated hemophilia clotting factors to patients with this illness.
Enter the 21st century. Progress has gone beyond perfecting recombinant technology. Now we have researchers trying to make hybrid molecules with longer half-lives and less immunogenicity. Alternate approaches to the coagulation cascade are being attempted to outdo the pathological aspects of hemophilia. Meanwhile, animal models continue to provide insight, impart enthusiasm, and serve as possible conduits for efficient production of coagulation factors. In one of a series of talks on hemophilia as part of the Education Session on Hemophilia, scheduled to take place today at 7:30 a.m. in Hall F3/F4 of the Orange County Convention Center, Dr. Steven Pipe will provide an overview of the seminal advances in the multipronged efforts to treat hemophilia. Be prepared to be astonished at how a select and small group of genetically redefined livestock can be marshaled to meet all of the annual factor requirements in the United States. Dr. Claire Philipp will discuss special problems concerning the older hemophilia patient, a unique and welcome prospect as better treatments help increase longevity. Dr. Robert Montgomery will talk about the several unique, ongoing, experimental and clinical trials that utilize gene transduction into different kinds of cells to express clotting factors in vivo. If you miss this morning’s session, it is taking place again tomorrow at 9:30 a.m. in the same location.
Not to forget the other well-known bleeding disorder — von Willebrand disease (VWD) — Monday’s oral session titled “Disorders of Coagulation or Fibrinolysis: von Willebrand” at 7:00 a.m. in Hall F3/F4 offers you new insights into this condition. Products to treat VWD are mostly plasma-derived; this has its own limitations. Dr. Tobias Suiter from Baxter BioScience will present a phase I study of the successful clinical use of recombinant von Willebrand factor (VWF) — the largest protein produced by recombinant technology (abstract #237). Appropriate treatment approaches to VWD, including prophylactic and anticipatory treatment, need to be further developed; Dr. Erik Berntorp from Malmö, Sweden, will present a large retrospective analysis of prophylactic administration of VWF concentrates to patients with severe VWD (abstract #236). Among 37 Swedish patients, bleeding episodes were reduced by 75 percent. These results are being evaluated in a prospective study and may assist us in better treating these patients. Also on Monday, Dr. Eva de Wee from Rotterdam, Netherlands, will present her group’s work on women with VWD during the oral session on “Disorders of Coagulation or Fibrinolysis: Inherited Bleeding Disorders” at 3:00 p.m. in Hall F3/F4 (abstract #542). Those with severe illness suffer from disabling menorrhagia and pronounced post-partum bleeding; 20 percent of all women with VWD undergo a hysterectomy. One hopes that simple and efficacious remedies are developed to assist these women in dealing with this disabling condition.
There is a great future for research in hemophilia and other bleeding disorders. This ASH meeting in Orlando, the city with the largest smiley face (Orlando breaks the world record for largest smiley face, Orlando Sentinel, October 10, 2010.) is sure to bring good news to patients, researchers, and physicians treating bleeding disorders.
Dr. Manchanda indicated no relevant conflicts of interest.
