Long-Term Safety and Effectiveness of Sebetralstat: Interim Analysis of KONFIDENT-S Open-label Extension.
Обработка статьи · этапы, ошибки и управление
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Farkas Henriette, Anderson John, Bouillet Laurence, Caballero Teresa, Cancian Mauro, Craig Timothy, Fukunaga Atsushi, Grivcheva-Panovska Vesna, Guilarte Mar, Honda Daisuke, Kanarek Henry, Kiani-Alikhan Sorena, Kinaciyan Tamar, Leguevaques Damia, Longhurst Hilary J, Magerl Markus, Manning Michael E, Martinez-Saguer Inmaculada, Melamed Isaac, O'Connor Maeve E, Peter Jonny, Savic Sinisa, Soteres Daniel F, Staevska Maria, Staubach Petra, Stobiecki Marcin, Tachdjian Raffi, Valerieva Anna, Yong Patrick F K, Hao James, Iverson Matthew, Smith Michael D, Yea Christopher M, Audhya Paul K, Aygören-Pürsün Emel, Bernstein Jonathan A, Cohn Danny M, Lumry William R, Riedl Marc A, Zanichelli Andrea, Maurer Marcus
Аннотация
OBJECTIVE: This analysis evaluated long-term safety and effectiveness of sebetralstat in KONFIDENT-S (NCT05505916), an ongoing, 2-year, open-label extension study.
METHODS: Enrolled participants aged ≥12 years with hereditary angioedema due to C1-inhibitor deficiency administered sebetralstat 600 mg for each attack, as early as possible, regardless of severity or location. The primary outcome was incidence of treatment-emergent adverse events (TEAEs). Secondary outcomes were times to beginning of symptom relief, reduction in severity, and complete resolution.
RESULTS: At data cutoff (January 31, 2024), 84 participants (mean age: 35.9 years; 14.3% <18 years; 64.3% female) treated 640 attacks with sebetralstat (84% of total attacks); the median (interquartile range [IQR]) number of attacks per participant was 5 (2-8). The median (IQR) time from attack onset to treatment was 9 (1-69) minutes. Baseline severity was as follows: 30.0% mild, 43.3% moderate, and 25.0% severe or very severe. Treatment-related TEAEs occurred in 8 participants (9.5%); none were serious. The median time to beginning of symptom relief (allowing for missing data entries between consecutive time points) was 1.68 (0.79-3.89) hours, to reduction in severity 6.57 (1.61 to >12) hours, and to complete attack resolution 21.02 (7.22 to >24) hours. No evidence of a diminished response over repeated treatments of attacks was observed.
CONCLUSIONS: Oral sebetralstat enabled compliance with treatment guidelines. No new safety signals were observed, and effectiveness for repeated attacks was consistent with the KONFIDENT trial results.