Baxter International Inc. (NYSE: BAX) today announced data featuring ADVATE [Antihemophilic Factor (Recombinant), Plasma/Albumin-Free Method], a leading recombinant treatment available worldwide, during the 24th Annual Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Amsterdam, The Netherlands. The first study identifies a correlation between peak levels of factor VIII (FVIII) in the body and the efficacy of treatment, indicating that frequency of peaks may be important when choosing hemophilia treatments. A second presentation reinforces that for both hemophilia patients and parents, efficacy through reduction in bleeds considerably outweighs infusion convenience as the primary treatment priority.
Exploring the Importance of Managing Both Peaks and Troughs
New findings from a post-hoc analysis of the ADVATE prophylaxis study presented at ISTH (PB 4.39-1) indicate important insights on the efficacy of prophylaxis treatment by closely monitoring the levels of recombinant treatment in a patient's body.
The study examined ''peaks,'' the highest levels of factor in the body in the first hours after infusion. Prior studies focused on ''troughs,'' representing the lowest activity levels as the body metabolizes the treatment (typically the period just prior to the next dose), and targeted a trough level of >1% above baseline, but did not consider peak values.1
The analysis identified a potentially important relationship between higher peak values, as well as time spent above 30 and 40 percent FVIII levels, (the "hemostatically effective" non-hemophilic FVIII range) and efficacy for prophylaxis in hemophilia A patients. This adds to the body of knowledge that by monitoring how long the factor remains active in the body, clinicians may be able to design optimal individualized dosing regimens to reduce the frequency of bleeds.
''Efficacy in preventing bleeding is the number one reason for choosing a hemophilia treatment. This pharmacokinetic research suggests that the dosing frequency to manage peaks in addition to troughs may aid in maximizing treatment effectiveness,'' said Leonard A. Valentino, M.D., director, Rush Hemophilia and Thrombophilia Center and Section of Pediatric Hematology/Oncology, Rush University Medical Center, Chicago, and lead investigator of this study. ''The finding may have implications when considering regimens with less frequent infusions that offer fewer FVIII peaks and less time within the hemostatically effective range.''
Both Patients and Parents Seek Efficacy as Primary Treatment Goal
Additional research presented at ISTH (#PB 3.36-4) discusses early findings that achieving low annual bleed rates is the most critical goal for people living with hemophilia. In the survey of treatment preferences among 76 adults living with hemophilia and 86 parents of children with hemophilia in the United States, the respondents agreed that achieving zero bleeds per year was more important than reducing dosing frequency from three times per week to twice weekly. Further, both groups preferred a "track record of ten years of product safety over a product new to market," even one with less frequent dosing.
''Overall, these studies emphasize that with regard to hemophilia patient care regimens, treatment is simply not a 'one size fits all' approach, and individualized regimens can help optimize the effectiveness each patient achieves,'' said Anders Ullman, M.D., Ph.D., vice president of global research and development in Baxter's BioScience business. ''Meeting the needs of individual patients and raising the standard of care are the primary focus of Baxter's long-term commitment in hemophilia and our pursuit of a future where one day, every patient can live a life without bleeds.''
Baxter International Inc.