Rate of decline in FVC Z-score (mean; 95% CI) per annum | ||||||
Prior to treatment | P value | Post treatment | P value | Difference between pretreatment and post-treatment rate of decline | P value | |
Total group (n=20) | −0.58 (−0.75 to −0.41) | <0.001 | −0.25 (−0.46 to −0.03) | 0.02 | 0.33 (0.02 to 0.66) | 0.04 |
SMA type 2 (n=12) | −0.61 (−0.77 to −0.44) | <0.001 | −0.08 (−0.32 to 0.15) | 0.48 | 0.52 (0.19 to 0.86) | 0.002 |
SMA type 3 (n=8) | −0.61 (−1.09 to −0.12) | 0.01 | −0.50 (−0.91 to −0.09) | 0.02 | 0.11 (−0.63 to 0.85) | 0.77 |
Rate of decline in percent predicted FVC (mean; 95% CI) per annum | ||||||
2 years prior to treatment | P value | During first year of treatment | P value | Difference between pretreatment and post-treatment rate of decline | P value | |
Total group (n=20) | −5.94 (−7.74 to −4.14) | <0.001 | −2.56 (−4.82 to −0.30) | 0.03 | 3.38 (−0.02 to 6.78) | 0.05 |
SMA type 2 (n=12) | −5.86 (−7.60 to −4.11) | <0.001 | −1.03 (−3.55 to 1.50) | 0.42 | 4.83 (1.20 to 8.47) | 0.009 |
SMA type 3 (n=8) | −7.69 (−12.71 to −2.67) | 0.003 | −4.75 (−9.01 to −0.49) | 0.03 | 2.94 (−4.70 to 10.59) | 0.45 |
^{.}
SMA, spinal muscular atrophy.