New tool accurately measures quality of life in HAE, detects improvements
Researchers used data from clinical trial to test AE-QoL questionnaire
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The Angioedema Quality of Life Questionnaire (AE-QoL) is a reliable and effective tool for measuring the impact of hereditary angioedema (HAE) on patients’ day-to-day lives — and can also detect meaningful improvements when the genetic disease becomes better controlled.
That’s according to an analysis of data from OASIS HAE (NCT05139810), a Phase 3 clinical trial testing Dawnzera (donidalorsen), a now-approved preventive treatment for HAE. Researchers used data from the study, which led to Dawnzera’s U.S. approval, to evaluate the questionnaire’s ability to assess health-related quality of life — dubbed HRQoL by the team — among people with HAE who took part in the trial.
The results showed that emotional concerns were important to participants, and that the AE-QoL could tell the difference between patients whose HAE was well- or poorly controlled.
“The AE-QoL demonstrated strong reliability, validity, and ability to detect change, supporting its use as an important patient-reported outcome measure for evaluating HRQoL in patients with HAE,” the researchers wrote.
The study, “Psychometric validation of the Angioedema Quality of Life Questionnaire (AE-QoL) for hereditary angioedema,” was published in the Journal of Patient-Reported Outcomes. It was funded by Ionis Pharmaceuticals, which markets Dawnzera; the work was mainly done by IQVIA, a U.S. health information technology and clinical research company.
HAE is characterized by recurrent episodes of severe swelling that affect different parts of the body. Health-related quality of life reflects the impact of a disease on a person’s daily life, well-being, and ability to function.
In this study, the research team evaluated whether the AE-QoL is a reliable tool for measuring HRQoL in people with HAE. The team used data from participants in OASIS-HAE, which tested Dawnzera against a placebo.
Researchers used 6 months of trial data to assess AE-QoL
Validation of the AE-QoL focused on scores collected at the start of the trial and after 24 weeks, or nearly six months, and included reliability — whether the questionnaire produced consistent data — and validity, or whether it evaluated what it was intended to measure. The analysis also looked at responsiveness, or whether the measure could detect meaningful improvements over time.
Of the 90 children and adults with HAE in the study, 48, or slightly more than half, were female. The average age was 37, with participant ages ranging from 12 to 68. At the start of the study, the average AE-QoL total score was 46, which dropped after 24 weeks to 25.1, showing improvement. In the AE-QoL, higher scores indicate a greater disease burden.
Among the questionnaire’s four domains, fears and shame had the highest average score at the start of the study, suggesting that emotional concerns were particularly important, according to the researchers. Participants with poor disease control had worse total scores than those with well-controlled disease, showing that the AE-QoL can distinguish between poorly and well-controlled HAE, the team noted.
The AE-QoL scores were compared with other established measures for HAE: One was the Angioedema Activity Score over 28 days (AAS-28), which measures disease activity; another was the Angioedema Control Test (AECT), an assessment of disease control. Strong relationships between AE-QoL and these measures supported convergent validity, meaning the questionnaire agreed with other clinical tools for HAE, the researchers noted.
The AE-QoL also demonstrated criterion validity, meaning its scores were related to clinical outcomes. Individuals with more frequent episodes of swelling generally reported worse quality of life.
This study affirms the AE-QoL to be a reliable, valid, and appropriate instrument for assessing HRQoL in patients with HAE.
In addition, the AE-QoL was sensitive to change. Improvements in disease activity, disease control, and swelling were reflected by better scores, the data showed. The total score, as well as the functioning and fears/shame domains, were especially sensitive to health status, the researchers noted.
“This study affirms the AE-QoL to be a reliable, valid, and appropriate instrument for assessing HRQoL in patients with HAE,” the researchers wrote, concluding that the questionnaire can consistently assess how HAE affects daily living, emotional well-being, and functioning, while also detecting meaningful improvements or worsening of the disease over time.
The team noted, however, that their study had a “limited sample size [that] precluded subgroup analyses and may be insufficient to … assess [the] structural validity” of the measure. As such, “further research is needed, with larger [patient groups], to validate the AE-QoL across diverse groups of adolescents with HAE and consolidate findings of structural validity.”
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