Humate-P Approved To Prevent Excessive Bleeding In Patients With Von Willebrand Disease Undergoing Surgery
Von Willebrand Disease
Humate-P, CSL Behring's factor replacement therapy for the treatment of hemophilia A and von Willebrand disease, has been approved by the U.S. Food and Drug Administration to prevent excessive bleeding during and after surgery, in patients with severe VWD and mild to moderate VWD where the use of desmopressin is known or suspected to be inadequate.
The new indication approval follows the results of two separate clinical studies - one in Europe, another in the United States - that demonstrate the hemostatic efficacy and safety of Humate-P for VWD patients undergoing major, minor and oral surgical procedures.
"For individuals with VWD, the most common inherited bleeding disorder, even minor surgery can present serious risks and require special care," said Garrett E. Bergman, M.D., Senior Director Medical Affairs, U.S. Commercial Operations at CSL Behring. "Humate-P, which for years has been used successfully to treat spontaneous bleeding episodes in VWD patients, can now be administered to help prevent excessive bleeding in these patients during and after surgery."
In the U.S. clinical study, the safety and hemostatic efficacy of Humate-P were studied in 35 VWD patients undergoing surgery; in Europe, Humate-P was investigated in an additional 27 subjects. For each of the 62 patients, a pharmacokinetic assessment was performed several days before the surgical procedure to individualize the dosing of Humate-P. Across the studies, 44 of the 62 patients underwent surgeries that could be classified as major (including joint replacement, intracranial surgery, and hysterectomy). In 61 subjects, investigators classified the efficacy of Humate-P as either good or excellent. The most common adverse events in the clinical trials subjects were postoperative hemorrhage (35 events in 19 subjects), postoperative nausea, and postoperative pain.
In 1999, the FDA approved Humate-P for use in treating severe VWD and mild to moderate VWD where the use of desmopressin is known or suspected to be inadequate. Previously, it had been approved only for adult patients with hemophilia A.
Von Willebrand disease, the most common hereditary bleeding disorder in the United States, affects approximately 1 to 2% of the U.S. population. It is caused by a deficiency or abnormality of the von Willebrand factor, a protein in the blood that is necessary for normal blood clotting. Men and women are equally likely to be affected by VWD. VWD is classified by type of defect, ranging from Type I (the most common and mildest) to Type III (the least common and most severe).
Women with VWD are more likely than other women to experience heavy, prolonged menstruation. Other common symptoms of VWD include frequent nosebleeds and easy bruising. Bleeding can be mild or serious and can occur as a result of injury, or without an obvious cause. More serious symptoms include bleeding into joints and internal organs. An individual with any of the three types of VWD may require special care during dental procedures, surgery, and childbirth.
There is no "cure" for VWD, but different situations can be treated effectively in different ways. Specific treatments may include: desmopressin acetate, which, in patients with mild VWD, can release stored von Willebrand factor; von Willebrand factor replacement therapies such as Humate-P; and oral contraceptives, to reduce menstrual bleeding and to raise von Willebrand factor levels in the bloodstream.