Table 1.
Relative treatment effects of FVC and 6MWD (random effect NMA), primary analysis (49/52 weeks of follow-up for all RCTs)
| Relative treatment effects measured as mean differences (95% credible interval) | ||||
|---|---|---|---|---|
| Outcome: Force vital capacity % predicted. Minimum clinically important diference range: 2.5–4.8%* | ||||
| PBO | ALG | AVAL | CM | |
| PBO | 3.58 (-2.95, 10.13) | 6.01 (-3.40, 15.33) | 3.11 (-6.79, 13.00) | |
| ALG | -3.58 (-10.13, 2.95) | 2.43 (-4.22, 9.07) | -0.48 (-7.97, 6.98) | |
| AVAL | -6.01 (-15.33, 3.40) | -2.43 (-9.07, 4.22) | -2.90 (-12.91, 7.07) | |
| CM | -3.11 (-13.00, 6.79) | 0.48 (-6.98, 7.97) | 2.90 (-7.07, 12.91) | |
| Outcome: Six-minute walking distance in metres. Minimum clinically important diference range: 24 m to 57 m * | ||||
| PBO | ALG | AVAL | CM | |
| PBO | 24.68 (3.97, 45.65) | 53.55 (19.66, 87.31) | 19.29 (-17.43, 56.09) | |
| ALG | -24.68 (-45.65, -3.97) | 28.87 (1.74, 55.66) | -5.39 (-35.72, 25.58) | |
| AVAL | -53.55 (-87.31, -19.66) | -28.87 (-55.66, -1.74) | -34.26 (-74.80, 6.96) | |
| CM | -19.29 (-56.09, 17.43) | 5.39 (-25.58, 35.72) | 34.26 (-6.96, 74.80) | |
A negative symbol indicates that the treatment in the top row is less effective than the treatment in the first column. ALG, Alglucosidase; AVAL, Avalglucosidase; CM, Cipaglucosidase + Miglustat; PBO, placebo; FVC, forced vital capacity, 6MWD six-minute walking distance; *Sources for minimum clinically important diference ranges are reported in the Discussion