Table 1.
Source | SMA TYPE | MRC baseline (mean + SD or mean (range)) | Time from infusion/FU timing | N | MRC change |
---|---|---|---|---|---|
Nusinersen-treated cohorts | |||||
De Wel | 3 and 4 | 36.9 + 10.3 | 14 months | 15 | Mean: 2.53, 99% CI 0.18–4.88 |
Walter | 3 | 113.95 + 22.91 | 10 months | 16 | Mean: 4.55 |
Mosche-Lilie | 2 and 3 | Not reported | 12 months | 10 | Mean %: + 2.13% |
Untreated cohorts | |||||
Wjngaarde | 2A | Not reported | 12 months | 68 | Annual slope: − 0.73, 95% CI − 1.00; − 0.45 |
Wjngaarde | 2B | Not reported | 12 months | 50 | Annual slope: − 0.65, 95% CI − 0.89; − 0.40 |
Wjngaarde | 3A | Not reported | 12 months | 63 | Annual slope: − 0.84, 95% CI − 1.07; − 0.61 |
Wjngaarde | 3B | Not reported | 12 months | 40 | Annual slope: − 0.83, 95% CI − 1.13; − 0.54 |
Carter GT | 2 | Mean 2.3 + 0.6* | 10 years | 18 | Decade slope − 0.24 per muscle |
Piepers 2008 | 3B and 4 | Mean 294 + 12 | Mean 30 months (19–36 months) | 9 | Mean change: 0 |
Wadman | 1C, 2, 3, 4 | Not reported | 12 months | 180 | Annual slope: − 1 |
Werlauff | 2 | 29% (9–41) | Median 17 years (12–20 years) | 21 | Annual slope: − 0.22 (Upper limb), CI − 0.39; − 0.02 |
Otto 2020 | 2 and 3 | 142.7 + 41.6 | Mean 13.1 months (368–442 days) | 9 | Mean change: − 1.1 |
Key to table: *average grade for all muscles groups combined