Table 1.
Clinical and MRI features
| Treatment | Group | FU MRI | Pt | Sex | MSA | Age (ys) | Timing MRI from DO/btw MRIs (mo) | STIR%LB first/second | ΔSTIR | T1-LB first/second | ΔT1 | MRC BS/FU | CK onset/first MRI/FU end (UI/L) | No. of treatments | IVIG/delay (mo) | Disease outcome | Clinical outcome |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Untreated | |||||||||||||||||
| Early-BT | |||||||||||||||||
| Yes | P1 | F | HMGCR | 39 | 2/7 | 26/0 | − 100% | 4/4 | 0% | 52/60 | 7400/7400/78 | 3 | Y/6 | R | A | ||
| P2 | F | HMGCR | 34 | 1/10 | 46/4 | − 92% | 0/2 | + 0.6% | 54/60 | 8800/8800/79 | 3 | Y/5 | R | A | |||
| P3 | F | HMGCR | 74 | 6/9 | 49/0 | − 100% | 40/46 | + 1.9% | 44/53 | 10,500/10,500/800 | 1 | Y/6 | R | Mild prox weakness | |||
| P4 | F | SRP | 57 | 4/16 | 51/21 | − 60% | 15/110 | + 30.4% | 40/56 | 6000/6000/250 | 3 | Y/15 | I | Mild prox weakness | |||
| P5 | F | SRP | 78 | 1/4 | 37/0 | − 100% | 18/18 | 0% | 43/57 | 8000/8000/80 | 3 | Y/5 | R | Very mild prox weakness | |||
| P6 | F | – | 45 | 5/17 | 32/3 | − 92% | 11/15 | + 1.3% | 50/60 | 3250/3250/150 | 2 | N | R | A | |||
| No | P7 | M | HMGCR | 61 | 3 | 52 | 31 | 42/50 | 25,000/25,000/2000 | 1 | N | I | Mild prox weakness | ||||
| P8 | F | SRP | 83 | 2 | n.a | 30 | 48/58 | 3000/3000/95 | 2 | N | R | Very mild prox weakness | |||||
| P9 | M | – | 65 | 0 | 27 | 29 | 54/60 | n.a./n.a./290 | 1 | N | R | A | |||||
| P10 | F | – | 84 | 4 | 58 | 102 | 26/34 | 3000/3000/96 | 2 | N | I | Mod prox-dist-ax weakness | |||||
| P11 | F | – | 22 | 0 | 8 | 0 | 48/60 | 30,000/30,000/450 | 1 | N | R | A | |||||
| Treated | |||||||||||||||||
| Early-WT | |||||||||||||||||
| Yes | P12 | M | HMGCR | 69 | 3/23 | 15/0 | − 100% | 50/50 | 0% | 54/60 | 8200/8200/62 | 2 | N | R | A | ||
| P13 | M | SRP | 62 | 4/22 | 40/27 | − 32% | 31/54 | + 7.4% | 49/60 | 7700/7700/250 | 2 | Y/5–9 | R | Axial weakness | |||
| No | P14 | F | SRP | 66 | 5 | 21 | 36 | 43/60 | 4000/400/150 | 3 | Y/5 | R | A | ||||
| P15 | F | – | 83 | 3 | 14 | 62 | 48/52 | 2100/2100/25 | 1 | N | I | Mild prox weakness | |||||
| Late | |||||||||||||||||
| Yes | P16 | M | HMGCR | 76 | 53/30 | 4/1 | − 66% | 54/56 | + 0.6% | 49/58 | 5300/410/270 | 3 | Y/7 | R | Mild prox weakness | ||
| P17 | F | HMGCR | 30 | 330/15 | 0/0 | – | 162/162 | 0% | 46/46 | n.a./205/80 | 2 | N | R | Mild prox weakness | |||
| P18 | F | SRP | 62 | 66/27 | 2/4 | + 100% | 62/62 | 0% | 50/57 | 3600/170/150 | 3 | N | R | Mild prox weakness | |||
| P19 | F | SRP | 49 | 15/6 | 40/42 | + 6.5% | 45/62 | + 5.4% | 54/54 | 7300/5000/300 | 3 | N | S | Mild prox weakness | |||
| P20 | F | SRP | 53 | 181/210 | 0/0 | 0% | 178/180 | 0% | 48/48 | 3500/n.a./37 | 3 | Y/96 | R | Mild-mod prox weakness | |||
| P21 | F | – | 39 | 164/17 | 23/23 | 0% | 116/116 | 0% | 48/48 | n.a./n.a./n.a | 3 | N | S | Mild-mod prox-ax weakness | |||
| No | P22 | M | M | – | 58 | 9 | 14 | 52/60 | 1200/n.a./700 | 3 | Y/9 | R | A |
FU follow-up; DO disease onset; btw between; BS baseline; Y yes; N no; R remission; I improvement; S stabilization/refractory; A asymptomatic