Aptevo Therapeutics recently announced patient-reported data on individuals currently being treated with IXINITY®, the company’s recombinant factor IX product. IXINITY® is indicated for the control and prevention of bleeding episodes and for perioperative management in adults and children, 12 years of age or older, with hemophilia B. The results were presented at the Thrombosis and Hemostasis 2018 Summit of North America, held in on March 8-10, 2018 in San Diego, CA.
According to an Aptevo press release the study was designed to “generate a descriptive analysis” from patient experiences with IXINITY®. Patient-reported data was gleaned via a questionnaire which focused on both clinical experiences and quality of life issues.
Investigators reported overall high satisfaction ratings based in part on low annualized bleeding rates and low impact on quality of life. Specifically, 89% of patients reported that they were “very satisfied” or “somewhat satisfied” with the product. Since starting IXINITY, a majority of patients reported that they were “very active” (22%) or “somewhat active” (56%). Lastly, a majority of patients receiving IXINITY® reported “no problems” or “slight problems” related to quality of life measures such as mobility, self-care, usual daily activities, pain or discomfort, anxiety or depression. Study limitations included a small sample size, potential for selection bias, recall bias among participants and the use of non-validated outcomes measures.
“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,” said Dr. Scott Stromatt, Chief Medical Officer for Aptevo. “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. 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,” said Dr. Stromatt.
Source: Aptevo press release dated March 12, 2018