scholarly journals P.025 Efficacy and safety results of the avalglucosidase alfa phase 3 COMET trial in participants with late-onset Pompe disease (LOPD)

2021 ◽  
Vol 48 (s3) ◽  
pp. S27-S27
Author(s):  
M Tarnopolsky ◽  
S Attarian ◽  
J Borges ◽  
F Bouhour ◽  
Y Choi ◽  
...  
Keyword(s):  
Muscle Strength ◽  
Neutralizing Antibodies ◽  
Late Onset ◽  
Primary Objective ◽  
Absolute Difference ◽  
Phase 3 ◽  
Alglucosidase Alfa ◽  
Related Quality ◽  
Health Related ◽  
Treatment Naïve

Background: Phase 3 COMET trial (NCT02782741) compares avalglucosidase alfa (n=51) with alglucosidase alfa (n=49) in treatment-naïve LOPD. Methods: Primary objective: determine avalglucosidase alfa effect on respiratory muscle function. Secondary/other objectives include: avalglucosidase alfa effect on functional endurance, inspiratory/expiratory muscle strength, lower/upper extremity muscle strength, motor function, health-related quality of life, safety. Results: At Week 49, change (LSmean±SE) from baseline in upright forced vital capacity %predicted was greater with avalglucosidase alfa (2.89%±0.88%) versus alglucosidase alfa (0.46%±0.93%)(absolute difference+2.43%). The primary objective, achieving statistical non-inferiority (p=0.0074), was met. Superiority testing was borderline significant (p=0.0626). Week 49 change from baseline in 6-minute walk test was 30.01-meters greater for avalglucosidase alfa (32.21±9.93m) versus alglucosidase alfa (2.19±10.40m). Positive results for avalglucosidase alfa were seen for all secondary/other efficacy endpoints. Treatment-emergent adverse events (AEs) occurred in 86.3% of avalglucosidase alfa-treated and 91.8% of alglucosidase alfa-treated participants. Five participants withdrew, 4 for AEs, all on alglucosidase alfa. Serious AEs occurred in 8 avalglucosidase alfa-treated and 12 alglucosidase alfa-treated participants. IgG antidrug antibody responses were similar in both. High titers and neutralizing antibodies were more common for alglucosidase alfa. Conclusions: Results demonstrate improvements in clinically meaningful outcome measures and a more favorable safety profile with avalglucosidase alfa versus alglucosidase alfa. Funding: Sanofi Genzyme

scholarly journals Tofacitinib in Patients with Ulcerative Colitis: Health-Related Quality of Life in Phase 3 Randomised Controlled Induction and Maintenance Studies

2018 ◽  
Vol 13 (1) ◽  
pp. 139-140 ◽  
Author(s):  
Julian Panés ◽  
Séverine Vermeire ◽  
James O Lindsay ◽  
Bruce E Sands ◽  
Chinyu Su ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Ulcerative Colitis ◽  
Health Related Quality ◽  
Phase 3 ◽  
Related Quality ◽  
Health Related ◽  
Randomised Controlled

High Levels of Physical Fitness Are Associated With Better Health-Related Quality of Life in Women With Fibromyalgia: The al-Ándalus Project

2019 ◽  
Vol 99 (11) ◽  
pp. 1481-1494 ◽  
Author(s):  
Inmaculada C Álvarez-Gallardo ◽  
Alberto Soriano-Maldonado ◽  
Víctor Segura-Jiménez ◽  
Fernando Estévez-López ◽  
Daniel Camiletti-Moirón ◽  
...  
Keyword(s):  
Muscle Strength ◽  
Physical Fitness ◽  
Cardiorespiratory Fitness ◽  
Composite Score ◽  
Cross Sectional ◽  
Sf 36 ◽  
Related Quality ◽  
Health Related ◽  
Cross Sectional Design

Abstract Background Physical fitness is a marker of health and is associated with health-related quality of life (HRQoL). Identifying which components of physical fitness are associated with HRQoL in people with fibromyalgia may contribute to the development of more specific therapeutic strategies. Objective The 2 aims of this study were to examine the association of different components of physical fitness (ie, flexibility, muscle strength, speed and agility, and cardiorespiratory fitness) with HRQoL and to determine the extent to which any association between the components of physical fitness and HRQoL were of clinical relevance to women with fibromyalgia. Design A cross-sectional design was used. Methods This study included 466 women with fibromyalgia from southern Spain (Andalusia). The Senior Fitness Test battery and the handgrip test were used to assess physical fitness, and the 36-Item Short-Form Health Survey (SF-36) was used to assess HRQoL. Tender points, cognitive impairment, anthropometric measurements, and medication usage were also measured. First, multivariate linear regression was used to assess the individual relationship of each physical fitness test with the 8 dimensions of the SF-36. Second, a standardized composite score was computed for each component of physical fitness (flexibility, muscle strength, speed and agility, and cardiorespiratory fitness). A 1-way analysis of covariance to assess the differences in each of the 8 dimensions of the SF-36 across each physical fitness composite score was conducted. Forward stepwise regression was performed to analyze which components of physical fitness were independently associated with the SF-36 physical and mental component scales. Results Overall, higher levels of physical fitness were associated with higher levels of HRQoL (regardless of the SF-36 subscale evaluated). The effect sizes for HRQoL between participants with the lowest and the highest physical fitness levels ranged from moderate to large (Cohen d = 0.53–0.90). The muscle strength composite score was independently associated with the SF-36 physical component scale, whereas the flexibility composite score and cardiorespiratory fitness were independently associated with the SF-36 mental component scale. Limitations A limitation was that the cross-sectional design precluded the establishment of causality. Additionally, only women were included in the study, because fibromyalgia predominantly affects women. Conclusions High levels of physical fitness were consistently associated with better HRQoL in women with fibromyalgia; clinically relevant differences were demonstrated between those at extreme physical fitness levels. Muscle strength, flexibility, and cardiorespiratory fitness were independent indicators of HRQoL. These results warrant further prospective research on the potential of fitness to predict HRQoL in this population.

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