Hemophilia: Hope Is In Our Blood
Education and Advocacy New treatments for hemophilia are giving those with the disease a more fulfilling life.
Over three thousand Canadians live with hemophilia, a lifelong, inherited genetic disorder that affects the ability to form effective blood clots. For those affected, even the smallest injury or procedure can result in bleeding symptoms ranging from mild to life-threatening. Untreated, this bleeding can cause disability and early death. The good news is that effective treatments exist for most patients in Canada, but the treatment itself can be taxing for both the patients and their families.
“In Canada, we’re fortunate that the worst outcomes from hemophilia are not seen as often as before, because of the tremendous health system we have that provides replacement recombinant coagulation factors for patients to use at home by self-infusion, and the 25 specialized medical clinics across Canada that oversee their care,” explains Dr. Shannon Jackson of the Department of Medicine at the University of British Columbia, who oversees the Adult Provincial Bleeding Disorders program at St. Paul’s Hospital in downtown Vancouver. “The burden of treatment on patients, however, can be significant.”
The coagulation factors (VIII and IX), which replace the proteins absent in the blood of hemophiliacs, are delivered by intravenous infusion into the bloodstream, a process that must be repeated over the entire life of the patient. For most patients with the more common form of severe hemophilia, two to four infusions are required every week.
Completing the circle of care
Though this recombinant factor treatment is an improvement over the options available in the past, it can still present a burden for patients, especially the very young. Treating the disorder itself is, after all, only one part of a complete circle of care. “These patients have great medical, physical, social, and psychological needs that are not always as well supported as they could be in Canada due to underfunding of expert clinics,” says Dr. Jackson. “Maintaining and expanding the specialized treatment support network is vital in helping patients manage their burden of treatment throughout their lives.”
Hope on the horizon
Fortunately, advances in coagulation factor technology are extending the half-life of the treatment, and thus making it practical to reduce the number of infusions and lessen that burden. “In general, with an extended half-life factor VIII or IX product, in our clinic we’re finding that we can usually reduce the number of infusion days needed each week by at least one,” says Dr. Jackson. “For example, a patient who was self-infusing three times a week may now only need to infuse twice a week. That one infusion a week has a huge impact, especially for children. It’s a big deal for the patients and parents who are living this every day.”
In addition to these therapies, the future holds promise for continued improvement in hemophilia care, particularly for patients who develop inhibitors, a serious immune response to their coagulation factor therapy. According to Dr. Jackson, “there are innovations that will hopefully change the lives of many hemophilia patients for the better.”