Table 4 Change in 6MWD
From: The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review
Country/countries | Name of subgroup | First author & publication year | Sample size | Mean age | Mean 6MWD metres | 6MWD (sd) | Time point for follow up | Mean 6MWD metres @FU | 6MWD metres @FU (sd) |
|---|---|---|---|---|---|---|---|---|---|
Australia; Belgium; Canada; France; Germany; Italy; Israel; Spain; Sweden; UK; USA | Ambulatory > =5y | McDonald, 2013 [22] | 57 | 8.3 | 361.1 | 87.5 | 1y | 317.4 | 152.3 |
Ambulatory-Steroid treated, <7y | 6 | NR | 382.8 | 42.7 | 416.9 | 35.1 | |||
Ambulatory-Steroid treated, > = 7y | 34 | 356.1 | 93.6 | 297.1 | 154.5 | ||||
Ambulatory-Steroid-naïve, <7y | 8 | 366.3 | 63.5 | 341.4 | 163.9 | ||||
Italy | Ambulatory < =7y, Continuous steroids | Mazzone,2014 [74] | NR | NR | NR | 18.8 | 48.2 | ||
Ambulatory <350 m(6MWT), <7y | Pane, 2014 [34] | 9 | 5.8 | 307.9 | 3y | −49.3 | 173 | ||
Ambulatory ≥350 m(6MWT), <7y | 19 | 6.16 | 407.2 | 19.1 | 76.8 | ||||
Ambulatory <350 m(6MWT), ≥7y | 25 | 9.87 | 288.7 | −199.2 | 121.8 | ||||
Ambulatory ≥350 m(6MWT), ≥7y | 43 | 8.9 | 428.7 | −115 | 136.5 | ||||
Ambulatory-boys | 96 | NR | NR | 1y | −15.8 | 77.3 | |||
2y | −58.9 | 125.7 | |||||||
3y | −104.22 | 146.2 | |||||||
Mazzone,2014 [74] | 106 | 8.3 | 1y | −25.8 | 74.3 | ||||
Mazzone,2014 [46] | 113 | 8.2 | −22.7 | 81 | |||||
2y | −64.7 | 123.1 | |||||||
Ambulatory boys-None or intermittent steroids | Mazzone,2014 [74] | 51 | NR | 1y | −40.6 | 82.2 | |||
Ambulatory boys-Continuous steroids | 55 | −12.1 | 64.1 | ||||||
Ambulatory boys < =7y | 35 | −7.8 | 63.9 | ||||||
Ambulatory boys >7y | 71 | −42.3 | 73.9 | ||||||
Ambulatory boys < =7y, None or intermittent steroids | NR | −0.44 | 73.7 | ||||||
Ambulatory boys >7y, None or intermittent steroids | −66.4 | 77.7 | |||||||
Ambulatory boys >7y, Continuous steroids | −23.6 | 65.9 | |||||||
Italy; Belgium | Ambulatory ≥100 m | Pane, 2014 [42] | 191 | 7.9 | 378.06 | 74.13 | −10.19 | 69.33 | |
Ambulatory ≥100 m, <7y | 80 | 5.84 | 383.09 | 64.4 | 27.37 | 53.02 | |||
Ambulatory ≥100 m, >7y | 111 | 9.38 | 374.43 | 80.5 | −37.25 | 67.21 | |||
Ambulatory ≥100 m-Duplications | 15 | 8.6 | 420.26 | 85.3 | 4.37 | 54.14 | |||
Ambulatory ≥100 m-Point mutations | 44 | 7.5 | 393.62 | 70.53 | −0.84 | 53.6 | |||
Ambulatory ≥100 m-All deletions | 132 | 7.96 | 368.07 | 71.93 | −14.95 | 75.09 | |||
Ambulatory ≥100 m-Deletions eligible for skipping exon 44 | 18 | 8.2 | 398.16 | 65.28 | −11.78 | 54.89 | |||
Ambulatory ≥100 m-Deletions eligible for skipping exon 45 | 15 | 8.4 | 334.46 | 72.46 | −21.6 | 111.76 | |||
Ambulatory ≥100 m-Deletions eligible for skipping exon 46 | 7 | 7.3 | 335.3 | 85.47 | 0.83 | 24.51 | |||
Ambulatory ≥100 m-Deletions eligible for skipping exon 50 | 9 | 7.4 | 358.63 | 77.11 | −7.56 | 44.84 | |||
Ambulatory ≥100 m-Deletions eligible for skipping exon 51 | 27 | 7.7 | 362.66 | 62.26 | −21.59 | 76.33 | |||
Ambulatory ≥100 m-Deletions eligible for skipping exon 53 | 28 | 8.6 | 344.11 | 67.16 | −34.18 | 77.99 | |||
US | Ambulatory-boys | Henricson, 2012 [75] | 17 | NR | 352 | 87 | −73 | 135 | |
Henricson, 2013 [21] | 24 | 7.9 | 369.5 | 79.3 | −53.67 | 25.96 |