Table 2.
Changes in HFMSE, 6‐minute walk test, and RULM score (12 months vs. baseline) in the overall SMA III population, according to the patient onset, ambulatory status, and age groups.
| N | Baseline Mean Score (95%CI) | 12‐month Mean Score (95%CI) | Mean difference (95%CI) | p‐value | |
|---|---|---|---|---|---|
| HFMSE score | |||||
| All | 104 | 37.56 (36.98–38.13) | 38.74 (38.17–39.31) | 1.18 (0.37–1.99) | 0.004 |
| Type IIIA | 49 | 33.55 (32.49–34.61) | 34.57 (33.51–35.63) | 1.02 (−0.47–2.51) | 0.179 |
| Type IIIB | 55 | 41.13 (40.56–41.69) | 42.45 (41.89–43.02) | 1.33 (0.53–2.12) | 0.001 |
| Non‐ambulant | 43 | 19.79 (18.82–20.76) | 21.27 (20.27–22.28) | 1.48 (0.09–2.88) | 0.038 |
| Ambulant | 60 | 50.55 (49.85–51.25) | 51.61 (50.92–52.30) | 1.06 (0.08–2.04) | 0.034 |
| Pediatric | 59 | 43.05 (42.22–43.88) | 44.58 (43.73–45.44) | 1.53 (0.34–2.72) | 0.0123 |
| Adults | 45 | 30.75 (29.97–31.53) | 31.54 (30.79–32.30) | 0.79 (−0.29–1.87) | 0.148 |
| RULM score | |||||
| All | 100 | 29.33 (29.00–29.66) | 29.91 (29.58–30.24) | 0.58 (0.12–1.04) | 0.014 |
| Type IIIA | 44 | 26.52 (26.02–27.02) | 27.77 (27.27–28.27) | 1.25 (0.54–1.96) | 0.001 |
| Type IIIB | 56 | 31.53 (31.12–31.95) | 31.59 (31.17–32.01) | 0.05 (−0.53–0.64) | 0.857 |
| Non‐ambulant | 46 | 24.65 (24.16–25.15) | 25.61 (25.10–26.12) | 0.95 (0.24–1.66) | 0.0009 |
| Ambulant | 53 | 33.98 (33.54–34.42) | 34.30 (33.86–34.73) | 0.32 (−0.30–0.94) | 0.313 |
| Pediatric | 45 | 31.90 (31.40–32.39) | 33.15 (32.65–33.66) | 1.26 (0.55–1.97) | 0.0007 |
| Adults | 55 | 27.31 (26.91–27.71) | 27.38 (26.99–27.78) | 0.07 (−0.48–0.63) | 0.792 |
| RULM score (baseline RULM score <37) | |||||
| All | 73 | 26.49 (26.06–26.93) | 27.38 (26.95–27.82) | 0.89 (0.28–1.50) | 0.005 |
| Type IIIA | 39 | 25.17 (24.63–25.73) | 26.61 (26.06–27.17) | 1.44 (0.65–2.22) | 0.0005 |
| Type IIIB | 34 | 28.00 (27.33–28.67) | 28.26 (27.59–28.94) | 0.26 (−0.68–1.22) | 0.581 |
| Non‐ambulant | 43 | 23.62 (23.12–24.13) | 24.80 (24.28–25.33) | 1.18 (0.45–1.91) | 0.002 |
| Ambulant | 29 | 31.61 (30.80–32.41) | 32.21 (31.43–32.99) | 0.60 (−0.52–1.72) | 0.289 |
| Pediatric | 31 | 29.52 (28.82–30.22) | 31.41 (30.68–32.13) | 1.88 (0.87–2.90) | 0.0004 |
| Adults | 42 | 24.38 (23.87–24.88) | 24.62 (24.12–25.11) | 0.24 (−0.47–0.94) | 0.505 |
| 6MWT score | |||||
| All | 51 | 321.00 (311.29–330.71) | 327.65 (317.94–337.35) | 6.65 (−7.08–20.37) | 0.339 |
| Type IIIA | 18 | 279.89 (264.90–294.88) | 269.83 (254.84–284.82) | −10.06 (−31.25–11.14) | 0.341 |
| Type IIIB | 33 | 343.42 (330.98–355.87) | 359.18 (346.74–371.63) | 15.76 (−1.84–33.36) | 0.078 |
| Pediatric | 34 | 319.87 (306.92–332.82) | 330.20 (316.85–343.55) | 10.32 (−8.28–28.93) | 0.272 |
| Adults | 17 | 323.03 (308.23–337.83) | 323.55 (309.55–337.55) | 0.52 (−19.85–20.89) | 0.959 |
Dark grey shaded cells: statistical significance.