Long-term Haegarda Found to Improve Quality of Life in HAE Patients

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Long-term treatment with the approved therapy Haegarda leads to improved quality of life in people with type 1 or 2 hereditary angioedema (HAE), new clinical trial data show.

The findings were published in the Orphanet Journal of Rare Diseases, in a study titled “Long-term health-related quality of life in patients treated with subcutaneous C1-inhibitor replacement therapy for the prevention of hereditary angioedema attacks: findings from the COMPACT open-label extension study.” The study was funded by CSL Behring, which makes Haegarda.

HAE types 1 and 2 are caused by the lack of a functional C1-inhibitor (C1-INH) protein, which ultimately causes swelling. Haegarda, which can be self-administered, is a C1-INH replacement medication — it contains plasma-derived C1-INH — that can be given by under-the-skin (subcutaneous) injection.

It’s designed to prevent HAE attacks.

In the U.S., Haegarda is approved for the preventive (prophylactic) treatment of HAE in individuals ages six and up. The medication also recently became available in Quebec, Canada.

The safety and efficacy of Haegarda were demonstrated in the COMPACT Phase 3 clinical trial (NCT01912456), which showed that the treatment could substantially reduce the rate of swelling attacks in people with HAE types 1 and 2.

Participants who completed COMPACT could enroll in its open-label extension study (NCT02316353); this extension study also enrolled some people who had not participated in the original COMPACT study.

Overall, 126 people with type 1 or 2 HAE were involved in the extension. Half of the participants received Haegarda at a dose of 40 international units per kg (IU/kg) twice weekly, while the rest were given a dosage of 60 IU/kg twice weekly. Of note, 60 IU/kg is currently the approved dose.

Most of the participants were female (60.3%), white (96.0%), and had type 1 HAE (89.7%). Their mean age was 40.5, with 10 participants between the ages of 8 and 16.

Previously-reported results from the extension study demonstrated that the treatment reduced the rate of swelling attacks by 97-98% in children and adults.

In the new study, researchers from CSL Behring and other institutions analyzed the treatment’s effect on quality of life measures.

Prior to the extension trial, the participants’ scores on the European quality of life-5 dimensions Questionnaire (EQ-5D) — a subjective assessment of health — were slightly higher (better) than in the general U.S. population.

By the time of the last study visit, which came after about a year for most participants, the scores on the EQ-5D in the 60 IU/kg dose group had significantly increased, indicating better overall health. Scores in the 40 IU/kg group also increased, but the difference was not statistically significant.
Scores on the depression portion of the Hospital Anxiety and Depression Scale (HADS) decreased significantly in the 60 IU/kg dose group, indicating that the treatment eased depression symptoms. Depression scores also decreased in the lower dose group, but the change was not significant.
On the anxiety portion of HADS, scores decreased significantly in both dosage groups, indicating that Haegarda lessened anxiety.
Treatment with both dosages also significantly decreased activity impairment — being unable to do activities, particularly in the context of work — as assessed by the Work Productivity and Activity Impairment questionnaire (WPAI). In the 60 IU/kg group, WPAI scores also significantly improved for presenteeism, which is being able to go to work, but being unable to fully do your job, and work productivity loss.
Study participants generally reported high satisfaction with the treatment, as assessed with the Treatment Satisfaction Questionnaire for Medication (TSQM). These scores increased significantly from the beginning to the end of the study.
Across these measurements, many participants achieved a minimal clinically important difference — the smallest change in score that would be noticeable to the patient in their day-to-day life. About a quarter of participants had a clinically important improvement across EQ-5D, HADS, WPAI, and TSQM.
In a subset of participants with available data, beneficial effects also were seen on the disease-specific quality-of-life assessments angioedema quality of life (AE-QoL) and the hereditary angioedema quality of life (HAE-QoL).
Overall, there were “significant improvements from baseline [the trial’s start] at almost every measured HRQoL [health-related quality-of-life] outcome,” in the 60 IU/kg group, the researchers wrote.
“In patients with [HAE types 1 and 2] and frequent angioedema attacks managed long-term with open-label [Haegarda] 60 IU/kg twice weekly, a battery of HRQoL assessments revealed clinically meaningful and sustained improvements from baseline in overall quality of life, anxiety, depression, productivity, and satisfaction with therapy,” they concluded.