Real-world treatment patterns and burden-of-disease of sub-optimally controlled hereditary angioedema
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Notice bibliographique
Résumé
Background: Hereditary angioedema (HAE) is a rare disease characterized by unpredictable, recurrent subcutaneous or submucosal swelling that negatively impacts patients' health-related quality of life (HRQoL). Despite treatment goals aimed at achieving complete control of the disease and normalizing patients' lives, the disease remains poorly controlled for some patients. Objectives: To describe the demographic, clinical and treatment characteristics, as well as the HRQoL impairment of patients with sub-optimally controlled HAE type I/II per treating physician's judgment, focusing on understanding the factors influencing the burden of illness. Methods: A chart review was conducted at 32 HAE care centers across 18 European countries, Canada, and Israel between April 2022 and January 2023 in 214 patients aged ≥12 years with HAE type I/II sub-optimally controlled with on-demand treatment (ODT) and/or long-term prophylaxis (LTP). Patients receiving lanadelumab were excluded, as it was not yet widely available during the eligibility period. A cross-sectional survey at patient enrollment included the Angioedema Quality of Life (AE-QoL) and EQ-5D-5L questionnaires to assess the impact of HAE on HRQoL. Results: Patients with uncontrolled HAE had a mean (standard deviation [SD]) of 9.9 (13) attacks per year, with a mean (SD) duration of 1.9 (1) days per attack. During the one-year observation period, 50.5% of patients were on ODT only, 36.0% used LTP and ODT concurrently, 6.5% used LTP without ODT, and 7.0% were untreated. Attenuated androgens (AA; stanozolol and danazol) in LTP were used by 24.7% of patients, while tranexamic acid (TA) and C1-esterase inhibitor (C1-INH) replacement products were used for LTP by 9.8% and 6.5%, respectively. The mean (SD) AE-QoL total score was 44.4 (24.0), indicating a moderate level of impairment, with women experiencing worse HRQoL (total score of 50.9 [SD 24] vs 37.3 [SD 23] in men) [where the minimal clinically important difference is 6 points] [1]. HRQoL worsened with increasing attack rates, from 41.4 (SD 24.0) among patients with one to 5 attacks/year, still indicating moderate impairment in HRQoL, to 73.0 (SD 27.0) for patients with >40 attacks/year. Conclusions: Suboptimal disease control in HAE was associated with the use of ODT only, as well as LTP mainly with AA/TA. It imposes a substantial burden on patients' HRQoL, more particularly, but not exclusively, for those with frequent attacks and for women. The results suggest a need for improved HAE management.ClinicalTrial.gov study identifier NCT04957641.
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Prédiction distillée sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Apprise à partir de 10 348 étiquettes directes de Codex et de 10 348 étiquettes directes de Gemma. Le mode candidate est l'union des têtes enseignantes seuillées; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont ni des étiquettes humaines ni des étiquettes directes de modèles de pointe.
Scores Codex et Gemma par catégorie
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,000 | 0,000 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,001 | 0,000 |
| Bibliométrie | 0,001 | 0,001 |
| Études des sciences et des technologies | 0,000 | 0,000 |
| Communication savante | 0,000 | 0,000 |
| Science ouverte | 0,000 | 0,000 |
| Intégrité de la recherche | 0,000 | 0,000 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,001 | 0,000 |
Scores machine (provisoires)
Les deux têtes enseignantes du modèle étudiant, lues sur ce travail. Un score ordonne la base pour la relecture; il n'affirme jamais une catégorie, et le statut de validation accompagne chaque rangée tel quel.
Scores de référence d'un modèle non mature (critères de maturité non atteints, 7 itérations). Un score ordonne; il n'affirme jamais une catégorie.
score_only:v0-immature-baseline · tel quel depuis la passe de notation : score_only signifie que le nombre peut ordonner les travaux, et qu'aucune étiquette de catégorie n'en découle