KING OF PRUSSIA, Pa., Aug. 18 CSL Behring is providing a$1.2 million grant to fund a first-of-its kind, nationwide study that whencompleted could serve as the basis for determining optimal prophylaxistreatment for postpartum women with von Willebrand disease (VWD).Investigators anticipate that the study results will produce a betterunderstanding of the physiology of VWD in the postpartum period, whethertreatment is needed and, if so, for how long.
Von Willebrand disease (VWD) is the most common hereditary bleedingdisorder in the United States, and is caused by a deficiency or abnormality ofvon Willebrand factor in the blood. According to the U.S. Centers for DiseaseControl (CDC), von Willebrand disease affects 1 to 2 percent of Americans --as many as 2.8 million people -- more than half of whom are women. VWD occursin males and females. Women, though, may suffer severe health consequences,such as life-threatening bleeding following childbirth, if their condition isnot correctly diagnosed and managed. The National Heart, Lung and BloodInstitute (NHLBI), part of the National Institutes of Health (NIH) and theU.S. Department of Health and Human Services, recently issued the firstclinical guidelines in the U.S. for the diagnosis and treatment of VWD, citingissues specific to women, such as labor and delivery, as an ongoing researchneed and opportunity. For more information on the guidelines go towww.nhlbi.nih.gov/guidelines/vwd/index.htm.
"As a leader in the development of coagulation therapies, CSL Behring iscommitted to improving the quality of life for people with rare and seriousdiseases," said Robert Lefebvre, General Manager and Vice President of U.S.Commercial Operations at CSL Behring. "We hope that CSL Behring's support ofthis groundbreaking study will enable researchers to produce results thatclinicians around the world can use in treating women who have VWD after theygive birth."
In all women, von Willebrand factor (VWF) and factor VIII (FVIII) levelsincrease throughout the third trimester and then decrease to baseline withinone month postpartum. There is anecdotal evidence that in women with VWD,this physiologic postpartum decrease in VWF and FVIII levels can be excessive,with their levels reaching even lower than their normal baseline, placing themat a much higher risk of postpartum hemorrhage. The new NHLBI guidelinesstate that because coagulation factors return to pre-pregnancy levels within14-21 days, health care providers should be in close contact with women duringthis period.
"While we know that decreasing VWF levels following childbirth can resultin serious complications for women with VWD there are very little clinicaldata available to guide physicians in treating these patients," said PeterKouides, MD, a principal investigator of the study and Medical and ResearchDirector of the Mary M. Gooley Hemophilia Center in Rochester, NY, one of thefive sites where the study will be conducted. "By studying the patterns indeclining VWF levels, we hope to learn whether treatment following birth isneeded and the optimum duration of therapy."
The grant will be shared among Duke University Medical Center in Durham,North Carolina, the Mary M. Gooley Hemophilia Center in Rochester, New York,Robert Wood Johnson University Hospital in New Brunswick, New Jersey and twoadditional study sites. Andra James, MD, of Duke University Medical Centerwill serve as the study's principal investigator.
This prospective cohort study will compare changes in blood levels of vonWillebrand factor proteins postpartum between 40 women with and 40 womenwithout VWD. At periodic intervals in the immediate postpartum period, vonWillebrand Factor: Ristocetin cofactor activity (VWF:RiCoF), factor VIII(FVIII) and von Willebrand factor antigen (VWF:Ag) levels will be measured.The study will seek to determine how qu