During the 2020 Virtual Congress of the International Society on Thrombosis and Haemostasis (ISTH 2020), which took place from July 12-14, participants discussed the latest in treatments, research, and diagnostic tools relating to bleeding disorders. In a presentation given by Dr. Christoph Königs, MD, PhD, he discussed the efficacy of Eloctate for patients with hemophilia A. Following the results of two Phase 3 clinical trials, Eloctate was shown to prevent bleeding episodes in previously untreated pediatric patients. See the findings and the presentation abstract on the ISTH 2020 website.
As suggested by the name, hemophilia A is a subtype of the rare bleeding disorder hemophilia. Patients with hemophilia A lack factor VIII (FVIII), a clotting protein. Without it, the blood fails to clot properly, resulting in excessive and abnormal bleeding. Because it is an inherited, X-linked disorder, most patients are males. An estimated 20,000 U.S. citizens have hemophilia A.
Symptoms and severity vary, but may include:
- Frequent nose bleeds
- Infant irritability
- Large and easy bruising
- Digestive and urinary tract bleeding
- Joint pain and swelling
- Bleeding after injury, dental work, or surgery
- Prolonged menstruation or bleeding after childbirth
Learn more about hemophilia A here.
Eloctate (efmoroctocog alfa) is an anti-bleeding therapy created using recombinant DNA. In fact, it is one of the first recombinant DNA clotting FVIII therapies to stay in the body for extended periods of time, offering less invasive treatment and less frequent dosing. It works by connecting FVIII to part of an antibody.
Phase 3 PUPs A-LONG Trial
In the open-label PUPs A-LONG clinical trial, researchers analyzed the safety, tolerability, and efficacy of Eloctate for previously untreated patients with hemophilia A. The patients were all males under the age of 6 who had severe cases of hemophilia A. Although past studies examined the therapy’s efficacy in previously treated patients, analyzing its efficacy in this group marked PUPs A-LONG as the first of its kind.
Ultimately, the trial wanted to understand how children were affected after taking Eloctate for 10+ days. Demographics included:
- 80 patients were under the age of 1.
- 20 patients had a family history of FVIII inhibitors (and therefore a family history of hemophilia).
- 81 patients were immediately given Eloctate, while 20 were given Eloctate alongside an additional preventative treatment. Of the initial 81, 69 were eventually moved to the other group during the course of the study.
- 87 patients finished the study. Of these, 68 patients took Eloctate for at least 50 days (approx. 1.75 months).
Researchers found that Eloctate use actually encouraged some patients to develop FVIII inhibitors in their blood. To get to this point took around 9 days. The additional use of preventative treatment reduced the risk of bleeding episodes, as did a single infusion of Eloctate.
Admittedly, there were some negative side effects, including falling, headaches or head injuries, flu-like symptoms, and fever. Over 1/4 of patients experienced a severe reaction such as hemorrhaging or thrombosis (blood clot formation). Despite this, Eloctate was found to be well-tolerated and safe overall.
Check out the source article here.