Hemophilia B Patients Report Positive Experience with Ixinity Treatment, Aptevo Says

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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HemACTIVE survey

Patients with hemophilia B are reporting a mostly positive response to treatment with Ixinity (recombinant coagulation factor IX), according to new data from Aptevo Therapeutics, the therapy’s maker.

The company presented the patient-reported outcomes data at the recent Thrombosis and Hemostasis 2018 Summit of North America in San Diego. The event is organized by THSNA Societies, a group of 15 leading nonprofit organizations for hemostasis and thrombosis in North America.

Data were from a study analyzing patient experiences with Ixinity, a recombinant coagulation factor IX designed for the control and prevention of bleeding episodes and for perioperative management in hemophilia B patients 12 years of age and older.

Researchers used a questionnaire filled out by patients to assess several clinical and quality of life measurements. Overall, patients reported a high level of satisfaction with low annualized bleed rates (ABR) and low impact on quality of life scores.

More specifically, 89 percent of patients reported they were somewhat or very satisfied with Ixinity. Since starting the treatment, 22 percent of patients reported they were very active, and 56 percent reported being somewhat active.

The median reported annualized bleed rate among patients on Ixinity for preventive treatment was 1.6, which is consistent with the median ABR observed during a previous pivotal clinical trial (1.52). Most patients also reported no problems or only slight problems across all areas of quality of life, including mobility, self-care, pain or discomfort, anxiety, or depression.

“Anecdotal reports from patients who have switched their factor IX therapy to Ixinity appear to suggest some clinical and quality of life benefit associated with Ixinity therapy,” Scott Stromatt, MD, chief medical officer of Aptevo, said in a press release. “We undertook the current outcomes study to try and gain a better understanding of how Ixinity was being used in the real world setting and to attempt to validate some of these anecdotal observations.”

Researchers noted several limitations of the patient-reported data, including a small sample size and a potential for selection bias and recall bias among participants, as well as the use of nonvalidated outcome measures.

“While the results must be interpreted with caution, as there are many limitations to this type of study, such as its small size and retrospective nature, we were intrigued to see trends supportive of a positive patient benefit with Ixinity.  As patients are known to respond to factor IX options differently, we see value in having a variety of options available for patients and look forward to building on the data set for Ixinity,” Stromatt said.

Ixinity has been approved for use in patients 12 and older by the U.S. Food and Drug Administration (FDA) since April 2015.

In April 2017, Aptevo presented interim data from a Phase 3 clinical trial evaluating Ixinity in patients under the age of 12, showing it was safe and effective in previously treated children with hemophilia B.

The information was presented at the 2017 Hemostasis and Thrombosis Research Society Scientific Symposium in Scottsdale, Arizona.