Treatment can be administered at the time of a bleed, in anticipation of an activity that might prompt bleeding (eg, sports), or on a regular basis (prophylaxis) to prevent bleeding.
Hemophilia treatment requires a team approach, involving a group of health care professionals who are experts in treating bleeding disorders, including hematologists, nurse specialists, physical therapists, social workers, and other medical professionals, such as dentists and orthopedic surgeons.
While there is no cure for hemophilia yet, several treatment options are available to patients, which involve infusing the missing clotting factor product directly into the vein. With proper treatment, bleeding stops when enough clotting factor reaches the bleeding site. It is critical to administer treatment as quickly as possible to prevent long-term damage.
Gene therapy is a novel approach to treatment. One type of gene therapy, called gene transfer, involves giving an individual with hemophilia a functional FVIII or FIX gene by an IV infusion. This approach to treatment, which holds the promise of eliminating the need for regular intravenous infusions of factor VIII of IX protein, may improve the patient experience.
Pfizer is currently collaborating with key research partners on other forms of gene therapy for hemophilia B, and other gene therapy research is underway at the company's Rare Disease Consortium.
Better treatment has improved the prognosis and increased the lifespans of patients with hemophilia. However, it is still important that they discuss how to maintain a healthy lifestyle with a health care professional. Some patients may fear exercise, thinking that it may cause bleeding, when, in reality, it can strengthen muscles to help protect against spontaneous bleeds and joint damage. The severity of a patient’s hemophilia must be considered when choosing any activity. With knowledge, healthy choices, and proper treatment, more people with hemophilia are playing an active role in managing their disease.
Work must still be accomplished in the developing world, where a large portion of patients with hemophilia are either not diagnosed or don’t have access to clotting factor treatment. Pfizer is working with the World Federation of Hemophilia (WFH) to improve the availability of medical care in the developing world through the WFH’s Twinning Program. Sponsored solely by Pfizer, this program pairs a hemophilia treatment center or patient advocacy organization in a developing country with a corresponding treatment center or patient organization in a developed one.