According to a story from BioSpace, a recent study revealed that hemophilia A patients were less likely to experience bleeds when they used new, extended half-life therapies on a preventive basis when compared to the use of standard half-life therapies in the same manner. These results have meaningful implications for patients and their physicians when it comes to decisions related to which treatments they opt to use.
Hemophilia is a genetic disorder which affects the ability of the blood to form clots, a process that is vital for stopping bleeding after a wound is sustained. The severity of symptoms can vary widely. The disorder is caused by a mutation found on the X chromosome. Symptoms include bleeding for a long time after an injury, risk of bleeding in the brain and joints, and easy bruising. Bleeding in the joints can cause permanent damage and brain bleeding can lead to headaches, decreased consciousness, and seizures. There are multiple types of hemophilia, with the most common types being type A and type B, which are distinguished by having deficiencies in different clotting factors. Treatment involves replacing the missing clotting factor. Drugs that thin the blood should be avoided. To learn more about hemophilia, click here.
Standard vs. Extended
Hemophilia A is the form of hemophilia which is linked to a deficiency affecting clotting factor VIII. Standard half-life drugs have been around for a while and are effective in managing bleeds for patients with hemophilia. However, they can negatively impact quality of life because patients may need infusions as much as four times in a week. The primary advantage offered by extended half-life treatments is that patients can infuse less frequently (as little as once per week) without compromising the effectiveness of the treatment. However, the results of this trial indicate that these drugs may in fact be more effective.
The research included 59 patients using standard half-life products and 50 that took extended half-life ones. Extended half-life products tend to be more expensive, but the study found that after factoring in copays and insurance, most patients only paid a fraction of the listed price. One extended therapy, Adynovate, was found not to be statistically more expensive than standard therapies.
While cost will always be a consideration for hemophilia A patients, these results suggest that extended half-life products offer greater effectiveness when compared to standard half-life products.