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- W4205771849 abstract "The efficacy of avalglucosidase alfa compared to alglucosidase alfa as measured by the EuroQol 5-dimension 5-level (EQ-5D-5L) from the phase 3 COMET trial in late-onset Pompe disease (LOPD) is presented. EQ-5D-5L, a multi-attribute utility measure of health-related quality of life, was collected in treatment-naïve LOPD patients. Change from baseline to week 49 in the five dimensions of EQ-5D-5L, including mobility, usual activities, self-care, pain/discomfort and anxiety/depression; the derived patient's health state/index (range: 0 to 1); and the visual analogue scale (VAS, self-rated health on a 0 [worst health] to 100 [best health]) were compared for the two treatment groups using Wilcoxon-Mann-Whitney test at the nominal level. One-hundred patients were randomized (51 to avalglucosidase alfa, 49 to alglucosidase alfa); 95.0% (51 [100%] avalglucosidase alfa, 44 [89.8%] alglucosidase alfa) completed the primary analysis period. Mean (SD) baseline EQ-5D-5L utility index scores in the avalglucosidase alfa and alglucosidase alfa groups were 0.58 (0.21) and 0.59 (0.22), respectively (higher is better), indicating a health status worse than the general population (general population ≥0.8; ≤0.5 requiring hospitalization). Mean (SD) changes from baseline to week 49 were greater with avalglucosidase alfa for mobility (-0.50 [0.89] vs. -0.14 [0.68], p<0.05), usual activities (-0.34 [0.89] vs. 0.00 [0.73, p<0.05), EQ-5D VAS (8.80 [15.01] vs. -0.33 [16.13], p<0.01) and EQ-5D-5L index (0.08 [0.18] vs. 0.04 [0.16], not significant) compared to alglucosidase alfa. The most relevant domains in LOPD (mobility, usual activities) showed greater improvement with avalglucosidase alfa, consistent with the VAS; as expected, differences between treatment groups were not observed in self-care, pain/discomfort, anxiety/depression scores. Taken together, avalglucosidase alfa shows an additional therapeutic benefit over the current standard of care on the treatment relevant aspects of LOPD." @default.
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- W4205771849 date "2021-10-01" @default.
- W4205771849 modified "2023-09-26" @default.
- W4205771849 title "POMPE DISEASE" @default.
- W4205771849 doi "https://doi.org/10.1016/j.nmd.2021.07.228" @default.
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