A high level of health-related quality of life (HRQoL) is achievable for patients managing hereditary angioedema (HAE), a rare and serious genetic disorder, when effective treatment such as C1-inhibitor (INH) concentrate is available, according to a prospective patient assessment published recently in Allergy & Asthma Proceedings, the official journal of Regional, State & Local Allergy, Asthma and Immunology Societies (RSLAAIS) and the American Association of Certified Allergists (AACA). More than half of the HAE patients participating in the assessment reported feeling somewhat or much better with the availability of C1-INH concentrate, and more than 80 percent of participants indicated a more optimistic outlook on the future.
"The unpredictability, pain and potentially life-threatening nature of acute HAE attacks has been shown to dramatically impact patients’ health-related quality of life, but limited research has been conducted regarding their HRQoL while being treated for those attacks," said Timothy J. Craig, D.O., Professor of Medicine and Pediatrics at Penn State University in Hershey, Pennsylvania, and one of the study’s investigators. "Based on our findings, the availability of C1-INH for the treatment of acute attacks of HAE appears to provide patients with a better outlook on the future and a greater level of security in disease treatment."
For people living with HAE, episodes of edema, or swelling, in various body locations, can have a significant impact on QoL. Patients who have abdominal attacks of HAE can experience episodes of extreme pain, diarrhea, nausea and vomiting caused by swelling of the intestinal wall. HAE attacks that involve the face or throat can result in airway closure, asphyxiation and, if untreated, death.
Study Design and Key Findings
The prospective patient assessment included a subset of 28 participants enrolled in the International Multicenter Prospective Angioedema C1-Inhibitor Trial (I.M.P.A.C.T) 2 study. During their participation in the study, participants had the opportunity to complete a short form (SF) 12, a multipurpose generic measure of HRQoL, every three months. The QoL impact of the availability of C1-INH concentrate was also evaluated.
Of the 28 patients participating in the survey, 18 subjects completed 60 quarterly SF-12 questionnaires, and 13 submitted SF-12 assessments for a time period of longer than three months. Results from the survey showed mean scores ranging from 44.8 to 93.2, with only one participant reporting a score less than 50. Mean SF-12 scores among the four participants with the greatest number of treated attacks (47 – 106 attacks per subject) ranged from 70.6 to 82.7. When questioned on the impact of having C1-INH readily available, the majority of patients reported feeling much or somewhat better compared to prior experience without it.
About I.M.P.A.C.T. 2
Findings of I.M.P.A.C.T. 2 were based on treatment with 20 U/kg bodyweight of C1-INH in 1,085 episodes of HAE attacks at any body location in 57 patients. The main study end-points were time to onset of symptom relief, time to complete resolution of all symptoms and safety. No drug-related serious adverse events were reported, nor were any rebound effects observed following C1-INH administration.
About CSL Behring
CSL Behring is a global leader in the plasma protein biotherapeutics industry. Passionate about improving the quality of patients' lives, CSL Behring manufactures and markets a range of safe and effective plasma-derived and recombinant products and related services. The company's therapies are used in the treatment of immune deficiency disorders, hereditary angioedema, hemophilia, von Willebrand disease, other bleeding disorders and inherited emphysema. Other products are used for the prevention of hemolytic diseases in the newborn, in cardiac surgery, organ transplantation and in the treatment of burns. The company also operates one of the world's largest plasma collection networks, CSL Plasma. CSL Behring is a subsidiary of CSL Limited, a biopharmaceutical company with headquarters in Melbourne, Australia. For more information, visit www.cslbehring.com.
Sheila A. Burke
Director, Communications & Public Relations
Worldwide Commercial Operations