The USC-affiliated study Hemophilia Utilization Group Study (HUGS) is collecting prospective data from six hemophilia treatment centers nationwide to provide an accurate estimate of treatment costs.
“Many insurers underestimate the lifetime cost of treatment, which was projected as $140,000 per person in 1995,” said Denise Globe, associate professor of pharmaceutical economics and policy at the USC School of Pharmacy and primary investigator for HUGS. “With the advent of new therapies, the cost has changed over the last few years.”
Today, advanced treatments—clotting factors that help prevent or stop bleeding—have become the standard of care. But these treatments have come with higher price tags.
“The study looks at the utilization of treatment options, as well as the costs associated with hospitalizations and missed days from work,” said Globe. “An objective is to provide insurers with data that quantifies the cost of care to manage the illness.”
Hemophilia is an inherited disease that is characterized by a lack of certain proteins in the blood-clotting system. These proteins—called clotting factors—protect the body from extreme blood loss due to injury.
“A common misconception is that hemophilia causes a person to bleed profusely when their skin is broken,” said Globe. “Instead, patients with hemophilia can spontaneously bleed into their cranium or major joints, including elbows, knees or ankles. Every time they bleed into a joint it becomes inflamed and is damaged over time.”
“Because women inherit an X chromosome from both their mother and father, they are much less likely to develop hemophilia A or B, “ said Globe. “Most women who inherit the defective gene are carriers and do not exhibit symptoms.”
Treatment for hemophilia patients has changed within the last five years, according to Robert Miller, physician assistant and assistant professor of clinical pediatrics of the Hemostasis and Thrombosis Center at Childrens Hospital Los Angeles.
“We used to treat patients ‘on demand,’ but they had difficulties recognizing when a bleed had occurred,” said Miller. “Today, about 50 percent of pediatric patients receive preventive infusions of clotting factor anywhere from two to three times a week to prevent bleeds. “The goal is to protect their joints to eliminate problems later in life.”
HUGS data is collected at hemophilia treatment centers at these institutions: University of California, San Francisco; Children’s Hospital of Orange County; Childrens Hospital Los Angeles; University of Texas at Houston; UMass Memorial Hospital and the University of Colorado Health Sciences Center.
HUGS in funded in part by the Federal Hemophilia Treatment Centers/Region IX.