Current and Future Hemophilia Treatment Options Based on the 15th Annual Hemophilia Research Study Update

In the last few years, great advances have been made in the medical management of patients with hemophilia. Nevertheless, there are still several challenges that remain, and new challenges, such as the contamination of blood products, that continue to emerge and require resolution. These challenges emphasize the need for an interdisciplinary evaluation of current treatment practices.

A diverse group of hemophilia and infectious diseases experts met at the 15th Annual Hemophilia Research Study Update meeting in January 2005 in San Juan, Puerto Rico, to discuss new results in treating and preventing complications in hemophilia, with particular focus on current clinical trials. Topics ranged from the significant shortcomings of current therapies to evolving solutions that may better control several significant challenges, such as the management of patients with inhibitors. The information in this supplement is based on the wide range of presentations and debates that took place during the meeting.

The first article reviews the blood-borne pathogen risk and discusses emerging pathogens and infectious disease risk. Due to mutability and cross-species transmission, the always prevalent concern about rapid global spread of newly emerging pathogens is an ongoing issue that affects blood supply and requires persistent foresight to prevent transmission and contain outbreaks of disease.

The second article clarifies our present understanding of the genetic and environmental factors that affect inhibitor development. The authors discuss studies that offer insights into the risk factors that will help decrease inhibitor development and improve treatment outcomes.

The next article, in two parts, focuses on current and future approaches to inhibitor management and aversion. Part one discusses developments in improving immune tolerance induction and experimental approaches to inhibitor eradication. Part two reviews current issues and management of inhibitor patients as well as strategies to further guide treatment in special patients, such as those with refractory bleeding events, those undergoing surgery, and those with acquired hemophilia.

The next article illustrates challenges and approaches to venous access in children, and the efficacy and tolerance of coagulation factor treatment in pediatric patients compared with adults. It offers some promising approaches in managing the pediatric patient and the adolescent/adult transition. The consensus of the group was that this challenging patient group needs to be included more often in additional studies to define optimal treatment and dosage.

The article that follows examines the pathophysiology of joint diseases and potential alternatives to replace factor (F) VIII concentrates, and discusses other possible mechanisms that underlie hemophilic synovitis. The experimental findings that are discussed in this article may create the groundwork for clinically relevant innovations to come.

The final article provides an overview of educational opportunities that will aid in developing the next generation of hemophilia treatment specialists. This is a timely report, given the increasing demand for experts in the many therapeutic developments in hemophilia and related disorders.

This supplement provides hemophilia treatment physicians with an overview of methods to enhance therapeutic results while minimizing complications associated with uncontrolled bleeding. I would like to thank Baxter BioScience (Westlake Village, CA) for their support of this program.