Advances in therapy · 2025
An Indirect Treatment Comparison of Avalglucosidase Alfa versus Cipaglucosidase Alfa Plus Miglustat in Patients with Late-Onset Pompe Disease.
Сравнительное исследованиеСтатьяНазвание и аннотация: Перевод в очереди
Обработка статьи · этапы, ошибки и управление
Загрузка состояния…
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.
Roberts Mark E, Proskorovsky Irina, Guyot Patricia, Shukla Pragya, Thibault Nathan, Hamed Alaa, Pulikottil-Jacob Ruth, O'Callaghan Lasair, Pollissard Laurence
Аннотация
INTRODUCTION: No head-to-head studies comparing the efficacy of avalglucosidase alfa (AVA) with cipaglucosidase alfa + miglustat (Cipa+mig) have been conducted in patients with late-onset Pompe disease (LOPD). Two indirect treatment comparisons (ITCs) were conducted to estimate the effects of AVA versus Cipa+mig.
METHODS: ITCs were conducted using simulated treatment comparisons (STCs), adjusting for differences in prognostic factors and treatment effect modifiers. An analysis of patients who were naïve to enzyme replacement therapy (ERT-naive) used anchored STC with individual patient data (IPD) from the Phase 3 COMET (NCT02782741) study of AVA versus alglucosidase alfa (ALG) and aggregate data from patients who were ERT-naïve in the Phase 3 PROPEL (NCT03729362) study of Cipa+mig versus ALG + placebo. For patients who were ERT-experienced, an analysis used unanchored STC with IPD for AVA from the COMET open-label extension, and from NEO-1 (NCT01898364)/NEO-EXT (NCT02032524) studies, and aggregate Cipa+mig data from PROPEL and ATB200-02 (NCT02675465).
RESULTS: In patients who were ERT-naïve, the difference (95% confidence interval, CI) in mean change from baseline (CFB) at Weeks 49-52 in forced vital capacity percent predicted (FVCpp) showed a numerical improvement for AVA versus Cipa+mig with values of 5.49% (- 0.87, 11.86) with mixed model repeated measures analysis (MMRM/MMRM) and 4.69% (- 3.22, 12.61) with MMRM/analysis of covariance (ANCOVA). For the 6-min walk test (6MWT), differences were 57.08 m (11.04, 103.12) with MMRM/MMRM and 41.88 m (- 5.46, 89.22) with MMRM/ANCOVA, the former being statistically significant (p < 0.02) and the latter numerically in favour of AVA. In patients who were ERT-experienced, at Weeks 48-52 differences for AVA versus Cipa+mig with MMRM/MMRM (CIs/p values unavailable) were 1.40% for FVCpp and 18.85 m for 6MWT; with MMRM/Mean CFB, differences of 1.16% (- 1.88, 4.19) for FVCpp and 7.67 m (- 21.67, 37.02) for 6MWT, indicating a numerical improvement in favour of AVA.
CONCLUSIONS: ITCs suggest more favourable respiratory and mobility outcomes with AVA versus Cipa+mig in patients with LOPD, regardless of prior ERT-experience.
METHODS: ITCs were conducted using simulated treatment comparisons (STCs), adjusting for differences in prognostic factors and treatment effect modifiers. An analysis of patients who were naïve to enzyme replacement therapy (ERT-naive) used anchored STC with individual patient data (IPD) from the Phase 3 COMET (NCT02782741) study of AVA versus alglucosidase alfa (ALG) and aggregate data from patients who were ERT-naïve in the Phase 3 PROPEL (NCT03729362) study of Cipa+mig versus ALG + placebo. For patients who were ERT-experienced, an analysis used unanchored STC with IPD for AVA from the COMET open-label extension, and from NEO-1 (NCT01898364)/NEO-EXT (NCT02032524) studies, and aggregate Cipa+mig data from PROPEL and ATB200-02 (NCT02675465).
RESULTS: In patients who were ERT-naïve, the difference (95% confidence interval, CI) in mean change from baseline (CFB) at Weeks 49-52 in forced vital capacity percent predicted (FVCpp) showed a numerical improvement for AVA versus Cipa+mig with values of 5.49% (- 0.87, 11.86) with mixed model repeated measures analysis (MMRM/MMRM) and 4.69% (- 3.22, 12.61) with MMRM/analysis of covariance (ANCOVA). For the 6-min walk test (6MWT), differences were 57.08 m (11.04, 103.12) with MMRM/MMRM and 41.88 m (- 5.46, 89.22) with MMRM/ANCOVA, the former being statistically significant (p < 0.02) and the latter numerically in favour of AVA. In patients who were ERT-experienced, at Weeks 48-52 differences for AVA versus Cipa+mig with MMRM/MMRM (CIs/p values unavailable) were 1.40% for FVCpp and 18.85 m for 6MWT; with MMRM/Mean CFB, differences of 1.16% (- 1.88, 4.19) for FVCpp and 7.67 m (- 21.67, 37.02) for 6MWT, indicating a numerical improvement in favour of AVA.
CONCLUSIONS: ITCs suggest more favourable respiratory and mobility outcomes with AVA versus Cipa+mig in patients with LOPD, regardless of prior ERT-experience.