Health-related quality of life in hereditary angioedema patients treated with subcutaneous C1 inhibitor (C1-INH) therapy in Canada
Bibliographic record
Abstract
BACKGROUND: Hereditary angioedema (HAE) therapies have demonstrated improvements in clinical symptoms and health-related quality of life (HR-QoL) in patients with HAE. Specific causes for improvement in HR-QoL have not been sufficiently explored in Canada. OBJECTIVE: To understand patient perspectives on the burden of HAE and their experiences taking subcutaneous plasma-derived C1 inhibitor concentrate (SC-pdC1INH), and to investigate changes in HR-QoL domains which include physical, mental, emotional, functional, social, and medical well-being. METHODS: A qualitative research design was employed using telephone interviews from May to July 2023 and a quantitative self-administered Angioedema QoL questionnaire (AE-QoL), which has a recall period of 4 weeks. A conceptual map was generated from the interviews that illustrated HR-QoL domains most important to patients. RESULTS: This study included 20 patients (50% female; mean age of 51 years; 85% HAE Type 1/2). Patients reported more controlled HAE since starting SC-pdC1INH, with fewer attacks (85%) and even no/nearly no attacks (60%) in the past year. Patients reported emotional benefits (reduced stress/anxiety regarding potential attacks [75%], and increased confidence in managing HAE [95%]). Positive impacts also included increased productivity/less missed days from work/school (40%) and improved physical ability/well-being (50%). The total mean AE-QoL Score was 34 (range 0-87; SD 24.9), indicating a small effect of HAE on HR-QoL in the 4-week pre-interview period. CONCLUSION: Overall, patients on long-term prophylaxis with SC-pdC1INH reported improvements in many HR-QoL domains, but most significantly relief of stress and anxiety associated with HAE, and improvement in social and physical well-being.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.001 | 0.002 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.000 |
| Bibliometrics | 0.001 | 0.002 |
| Science and technology studies | 0.003 | 0.001 |
| Scholarly communication | 0.001 | 0.000 |
| Open science | 0.000 | 0.001 |
| Research integrity | 0.000 | 0.001 |
| Insufficient payload (model declined to judge) | 0.001 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".