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. 2024 Apr 8;31(7):e16292. doi: 10.1111/ene.16292

TABLE 1.

Differences between good and bad responders.

Parameter Delta FVC ≥ 10% N Mean SD P‐value Delta 6MWT ≥ 10% N Mean SD P‐value
Demographics
Diagnosis age (years) Improvement 7 33.57 10.57 0.367 Improvement 6 38.83 17.62 0.336
Worsening 5 41.80 11.74 Worsening 4 48.50 17.79
Weight at switch (kg) Improvement 7 76.85 17.19 0.410 Improvement 6 75.60 10.25 0.437
Worsening 5 72.80 24.18 Worsening 4 67.67 19.66
ERT beginning age (years) Improvement 7 40.43 8.87 0.684 Improvement 6 43.00 15.95 0.331
Worsening 5 44.00 10.42 Worsening 4 52.50 19.84
ERT switch age (years) Improvement 7 51.00 10.83 0.745 Improvement 6 54.33 14.86 0.257
Worsening 5 52.80 13.88 Worsening 4 64.25 23.99
ERT duration before switch (years) Improvement 7 10.57 3.60 0.463 Improvement 6 11.33 4.89 1.000
Worsening 5 8.80 3.83 Worsening 4 11.75 4.65
Respiratory parameters
FVC at switch (L) Improvement 7 1.821 1.166 0.167 Improvement 6 1.883 1.179 0.548
Worsening 5 2.652 0.970 Worsening 3 2.027 0.256
FVC at switch (% of the theorical values) Improvement 7 43.6 16.7 0.010 Improvement 6 49.4 16.3 0.795
Worsening 5 79.8 25.3 Worsening 3 53.7 16.3
FVC in supine position (L) Improvement 2 1.195 0.629 0.333 Improvement 1 1.040
Worsening 2 2.640 0.622 Worsening 2 1.950 0.438
FVC in supine position (% predicted) Improvement 2 37.50 14.85 0.333 Improvement 1 29.00
Worsening 2 78.00 32.52 Worsening 2 59.00 15.56
Maximal inspiratory pressure (cmH20) Improvement 5 35.22 17.47 1.000 Improvement 5 48.05 35.71
Worsening 3 30.66 13.81 Worsening 1 33.00
Maximal inspiratory pressure (% of theorical values) Improvement 5 40.80 17.20 0.905 Improvement 5 55.53 35.69
Worsening 4 42.18 17.26 Worsening 1 37.00
Maximal expiratory pressure (cmH20) Improvement 5 48.05 24.95 0.857 Improvement 5 47.34 8.07
Worsening 2 37.46 16.33 Worsening 1 32.00
Maximal expiratory pressure (% of theorical values) Improvement 5 44.40 26.89 1.000 Improvement 5 48.72 9.26
Worsening 3 44.34 10.01 Worsening 1 20.00
Motor parameters
6MWT at switch (m) Improvement 4 100.25 91.46 0.533 Improvement 5 161.20 111.20 0.571
Worsening 2 245.50 181.73 Worsening 2 217.50 119.50
Motor functional measure dimension 1 Improvement 6 25.77 30.13 0.517 Improvement 5 30.26 32.40
Worsening 3 47.86 30.91 Worsening 1 66.67
Motor functional measure dimension 2 Improvement 6 76.78 11.53 0.154 Improvement 5 76.67 24.42
Worsening 3 89.81 6.99 Worsening 1 100.00
Motor functional measure dimension 3 Improvement 6 95.19 3.01 0.596 Improvement 5 95.44 5.43
Worsening 3 93.65 5.50 Worsening 1 100.00
Total motor functional measure Improvement 6 61.64 16.08 0.381 Improvement 5 61.88 22.60
Worsening 3 73.61 12.43 Worsening 1 86.46
Evolution parameters
deltaFVC (Y2–Y1) (L) Improvement 6 0.018 0.248 0.126 Improvement 6 0.113 0.227 0.697
Worsening 5 1.37 1.782 Worsening 3 0.377 0.49
slopeFVC (Y2–Y1) (%) Improvement 6 −0.6 14.20 0.120 Improvement 6 8.40 10.10 0.905
Worsening 5 43.8 51.70 Worsening 3 36.00 55.50
delta6MWT (Y2–Y1) (m) Improvement 3 47 11.79 0.400 Improvement 5 18.40 48.73 0.857
Worsening 2 10 41.01 Worsening 2 67.50 55.86
slope6MWT (Y2–Y1) (%) Improvement 2 40.70 0.30 0.667 Improvement 5 10.40 48.30 1.000
Worsening 2 8.40 0.19 Worsening 2 26.50 25.50
deltaFVC (Y1–Switch) (L) Improvement 7 0.179 0.220 0.383 Improvement 6 0.122 0.175 0.381
Worsening 3 0.397 0.337 Worsening 3 0.027 0.103
slopeFVC (Y1–Switch) (%) Improvement 7 7.80 10.80 0.383 Improvement 6 4.40 12.60 0.548
Worsening 3 16.60 14.30 Worsening 3 1.80 5.50
delta6mwt (Y1–Switch) (m) Improvement 3 126.41 90.41 0.800 Improvement 5 159.55 110.52 0.571
Worsening 2 271.62 186.53 Worsening 2 215.98 118.75
slope6mwt (Y1–Switch) (%) Improvement 3 98.10 2.00 0.800 Improvement 5 98.40 2.00 0.857
Worsening 2 99.10 0.04 Worsening 2 99.39 4.00
deltaFVC (Switch–Y+1) (L) Improvement 7 −0.393 0.226 0.003 Improvement 5 −0.23 0.352 0.556
Worsening 5 0.376 0.170 Worsening 4 −0.055 0.101
slopeFVC (Switch–Y+1) (%) Improvement 7 −26.43 18.95 0.006 Improvement 5 −13.20 18.46 0.461
Worsening 5 14.60 5.94 Worsening 4 −2.75 6.80
delta6MWT (Switch–Y+1) (m) Improvement 7 7.43 35.83 0.222 Improvement 6 −31.33 25.00 0.010
Worsening 2 9.50 0.71 Worsening 4 56.00 31.45
slope6MWT (Switch–Y+1) (%) Improvement 7 −1.29 13.756 0.500 Improvement 6 −28.00 28.66 0.014
Worsening 2 5.50 3.536 Worsening 4 21.75 6.85

Note Values in bold type represent significant differences. Positive values indicate worsening with time. N varies with missing data.

Abbreviations: 6MWT, Six‐Minute Walk Test; ERT, enzyme replacement therapy; FVC, forced vital capacity; SD, standard deviation; Y1, 1 year before the switch; Y+1, 1 year after the switch; Y2, 2 years before the switch.