Subcutaneous Haegarda as a routine prevention for attacks of hereditary angioedema (HAE) significantly improves patients’ quality of life, such as lower anxiety levels and increased productivity at work, researchers found.
The study, “Health-Related Quality-of-Life with Subcutaneous C1-inhibitor for Prevention of Attacks of Hereditary Angioedema,” was published in The Journal of Allergy and Clinical Immunology: In Practice.
HAE is a rare genetic disease characterized by recurrent episodes of severe swelling — angioedema — and pain. The most commonly involved areas of the body are the face, limbs, intestinal tract, and airways.
Hereditary angioedema is caused by low levels (HAE type 1) or improper function (HAE type 2) of a protein called C1-esterase inhibitor, or C1-INH. In these cases, the immune system and blood flow processes may become excessively activated, causing swelling.
HAE can have profound negative effects on several aspects of patients’ health-related quality of life (HRQoL), including anxiety and productivity. The unpredictability of HAE attacks and fear of asphyxiation due to airway swelling are frequently cited reasons for anxiety and a decreased quality of life.
Although some improvements have been noted following the appearance of HAE therapies with administration of C1-INH, patients continue to report negative impacts of the disease in their quality of life.
Therefore, preventing hereditary angioedema attacks would greatly contribute to improving the lives of HAE patients. Haegarda, developed by CSL Behring, is the first C1-INH for subcutaneous administration to prevent HAE attacks in adolescents and adults. The self-administered subcutaneous injections are a more convenient treatment than the previously approved intravenous treatments.
The COMPACT Phase 3 trial (NCT01912456) previously evaluated the effectiveness, safety, and tolerability of Haegarda in the prevention of HAE types 1 and 2.
Ninety patients were randomized to one of four treatment sequences: twice-weekly doses of 40 IU/kg or 60 IU/kg of C1-INH for 16 weeks, preceded or followed by 16 weeks of twice-weekly placebo injections. All HAE attacks were treated with on-demand treatment as necessary.
Results from the trial showed that Haegarda was safe and efficiently reduced the number of HAE attacks, compared with placebo.
Now, researchers evaluated the HRQoL in COMPACT trial participants. HRQoL assessments were performed at week 14 (final visit) through the use of the European Quality of Life-5 Dimensions Questionnaire (EQ-5D-3L), the Hospital Anxiety and Depression Scale (HADS), the Work Productivity and Activity Impairment Questionnaire (WPAI), the Treatment Satisfaction Questionnaire for Medication (TSQM), and the Investigator and Subject Global Assessments of Response to Therapy (IGART and SGART).
At week 14, Haegarda as a preventive treatment had improved many aspects of HAE burden, especially in anxiety, work productivity, and activity impairment, compared with placebo. Patients self-administering Haegarda as routine prevention also reported high levels of treatment satisfaction and effectiveness, compared to on-demand treatment alone (placebo injections).
At the same time, patients administering Haegarda treatment were seeing better clinical improvements than the ones on placebo.
The results “corroborate the objective clinical benefits observed in the COMPACT study and suggest that routine prevention of HAE attacks with C1-INH [subcutaneous] is a major advance in HAE management, improving anxiety and enabling patients with C1-INH-HAE to regain an active and productive lifestyle,” researchers wrote.
The study had some limitations, including the use of generic HRQoL tools instead of the recently validated HAE-specific tools (because they were not available at the time), and the short observation period of the trial. Additional real-world data is needed to confirm these results.
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