Table 1
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Summary table of survival statistics after the dataset was split according to type of treatment

Viral vectorsASOsSmall molecules
Total effect on survival per treatment type1.51 [1.28–1.74; P < 0.01]1.12 [0.99–1.26; P < 0.01]1.02 [0.85–1.20; P < 0.01]
Timing of treatment
 Subgroup differences?P < 0.01P < 0.01P = 0.07
 E2.13 [1.79–2.47; P = 0.28; k = 2]0.01 [−0.23–0.25; P = 0.46; k = 2]1.68 [0.35–3.01; P < 0.01; k = 3]
 P0/11.62 [1.35–1.88; P < 0.01; k = 49]1.20 [1.04–1.35; P < 0.01; k = 182]1.15 [−1.11–3.41; P < 0.01; k = 33]
 P21.21 [0.46–1.96; P < 0.01; k = 6]0.90 [0.53–1.26; P < 0.01; k = 33]0.74 [0.55–0.92; P < 0.01; k = 22]
 P3+0.92 [0.37–1.47; P < 0.01; k = 8]0.92 [0.57–1.27; P < 0.01; k = 13]1.10 [0.87–1.33; P < 0.01; k = 50]
Frequency of Administration
 Subgroup differences?P = 0.18P < 0.01N/A
 One dose1.47 [1.24–1.71; P < 0.01; k = 61]0.80 [0.64–0.97; P < 0.01; k = 120]N/A [k = 0]
 Multiple doses2.13 [1.20–3.06; P < 0.01; k = 4]1.48 [1.28–1.68; P < 0.01; k = 110]1.02 [0.85–1.20; P < 0.01; k = 77]
SMA model
 Subgroup differences?P < 0.01P < 0.01P < 0.01
 Severe Type I0.50 [−0.24–1.24; P = 0.03; k = 2]1.37 [0.97–1.77; P < 0.01; k = 39]0.00 [−0.36–0.36; N/A; k = 1]
 SMNΔ71.62 [1.36–1.88; P < 0.01; k = 53]0.75 [0.59–0.90; P < 0.01; k = 96]1.06 [0.85–1.28; P < 0.01; k = 58]
 TaiwaneseN/A [k = 0]1.51 [1.29–1.72; P < 0.01; k = 89]1.07 [0.68–1.46; P < 0.01; k = 9]
 Smn2B1.12 [0.73–1.51; P < 0.01; k = 10]N/A [k = 0]0.84 [0.36–1.32; P < 0.01; k = 9]
 BurgheronN/A [k = 0]0.33 [0.14–0.52; P < 0.01; k = 2]N/A [k = 0]
 OtherN/A [k = 0]0.26 [−0.69–1.20; P < 0.01; k = 4]N/A [k = 0]
Route of administration
 Subgroup differences?P < 0.01P < 0.01P < 0.01
 CNS1.73 [1.42 −2.04; P < 0.01; k = 33]0.62 [0.45–0.78; P < 0.01; k = 103]0.16 [−0.15–0.47; P = 0.21; k = 2]
 Systemic1.13 [0.83–1.42; P < 0.01; k = 29]1.53 [1.34–1.72; P < 0.01; k = 116]1.05 [0.87–1.22; P < 0.01; k = 75]
 Both2.83 [2.55–3.12; P = 0.47; k = 3]1.70 [1.32–2.08; P < 0.01; k = 11]N/A [k = 0]
Viral vectorsASOsSmall molecules
Total effect on survival per treatment type1.51 [1.28–1.74; P < 0.01]1.12 [0.99–1.26; P < 0.01]1.02 [0.85–1.20; P < 0.01]
Timing of treatment
 Subgroup differences?P < 0.01P < 0.01P = 0.07
 E2.13 [1.79–2.47; P = 0.28; k = 2]0.01 [−0.23–0.25; P = 0.46; k = 2]1.68 [0.35–3.01; P < 0.01; k = 3]
 P0/11.62 [1.35–1.88; P < 0.01; k = 49]1.20 [1.04–1.35; P < 0.01; k = 182]1.15 [−1.11–3.41; P < 0.01; k = 33]
 P21.21 [0.46–1.96; P < 0.01; k = 6]0.90 [0.53–1.26; P < 0.01; k = 33]0.74 [0.55–0.92; P < 0.01; k = 22]
 P3+0.92 [0.37–1.47; P < 0.01; k = 8]0.92 [0.57–1.27; P < 0.01; k = 13]1.10 [0.87–1.33; P < 0.01; k = 50]
Frequency of Administration
 Subgroup differences?P = 0.18P < 0.01N/A
 One dose1.47 [1.24–1.71; P < 0.01; k = 61]0.80 [0.64–0.97; P < 0.01; k = 120]N/A [k = 0]
 Multiple doses2.13 [1.20–3.06; P < 0.01; k = 4]1.48 [1.28–1.68; P < 0.01; k = 110]1.02 [0.85–1.20; P < 0.01; k = 77]
SMA model
 Subgroup differences?P < 0.01P < 0.01P < 0.01
 Severe Type I0.50 [−0.24–1.24; P = 0.03; k = 2]1.37 [0.97–1.77; P < 0.01; k = 39]0.00 [−0.36–0.36; N/A; k = 1]
 SMNΔ71.62 [1.36–1.88; P < 0.01; k = 53]0.75 [0.59–0.90; P < 0.01; k = 96]1.06 [0.85–1.28; P < 0.01; k = 58]
 TaiwaneseN/A [k = 0]1.51 [1.29–1.72; P < 0.01; k = 89]1.07 [0.68–1.46; P < 0.01; k = 9]
 Smn2B1.12 [0.73–1.51; P < 0.01; k = 10]N/A [k = 0]0.84 [0.36–1.32; P < 0.01; k = 9]
 BurgheronN/A [k = 0]0.33 [0.14–0.52; P < 0.01; k = 2]N/A [k = 0]
 OtherN/A [k = 0]0.26 [−0.69–1.20; P < 0.01; k = 4]N/A [k = 0]
Route of administration
 Subgroup differences?P < 0.01P < 0.01P < 0.01
 CNS1.73 [1.42 −2.04; P < 0.01; k = 33]0.62 [0.45–0.78; P < 0.01; k = 103]0.16 [−0.15–0.47; P = 0.21; k = 2]
 Systemic1.13 [0.83–1.42; P < 0.01; k = 29]1.53 [1.34–1.72; P < 0.01; k = 116]1.05 [0.87–1.22; P < 0.01; k = 75]
 Both2.83 [2.55–3.12; P = 0.47; k = 3]1.70 [1.32–2.08; P < 0.01; k = 11]N/A [k = 0]

K, number of comparisons per group. Values indicate effect size [95% CI].

Table 1
Open in new tab

Summary table of survival statistics after the dataset was split according to type of treatment

Viral vectorsASOsSmall molecules
Total effect on survival per treatment type1.51 [1.28–1.74; P < 0.01]1.12 [0.99–1.26; P < 0.01]1.02 [0.85–1.20; P < 0.01]
Timing of treatment
 Subgroup differences?P < 0.01P < 0.01P = 0.07
 E2.13 [1.79–2.47; P = 0.28; k = 2]0.01 [−0.23–0.25; P = 0.46; k = 2]1.68 [0.35–3.01; P < 0.01; k = 3]
 P0/11.62 [1.35–1.88; P < 0.01; k = 49]1.20 [1.04–1.35; P < 0.01; k = 182]1.15 [−1.11–3.41; P < 0.01; k = 33]
 P21.21 [0.46–1.96; P < 0.01; k = 6]0.90 [0.53–1.26; P < 0.01; k = 33]0.74 [0.55–0.92; P < 0.01; k = 22]
 P3+0.92 [0.37–1.47; P < 0.01; k = 8]0.92 [0.57–1.27; P < 0.01; k = 13]1.10 [0.87–1.33; P < 0.01; k = 50]
Frequency of Administration
 Subgroup differences?P = 0.18P < 0.01N/A
 One dose1.47 [1.24–1.71; P < 0.01; k = 61]0.80 [0.64–0.97; P < 0.01; k = 120]N/A [k = 0]
 Multiple doses2.13 [1.20–3.06; P < 0.01; k = 4]1.48 [1.28–1.68; P < 0.01; k = 110]1.02 [0.85–1.20; P < 0.01; k = 77]
SMA model
 Subgroup differences?P < 0.01P < 0.01P < 0.01
 Severe Type I0.50 [−0.24–1.24; P = 0.03; k = 2]1.37 [0.97–1.77; P < 0.01; k = 39]0.00 [−0.36–0.36; N/A; k = 1]
 SMNΔ71.62 [1.36–1.88; P < 0.01; k = 53]0.75 [0.59–0.90; P < 0.01; k = 96]1.06 [0.85–1.28; P < 0.01; k = 58]
 TaiwaneseN/A [k = 0]1.51 [1.29–1.72; P < 0.01; k = 89]1.07 [0.68–1.46; P < 0.01; k = 9]
 Smn2B1.12 [0.73–1.51; P < 0.01; k = 10]N/A [k = 0]0.84 [0.36–1.32; P < 0.01; k = 9]
 BurgheronN/A [k = 0]0.33 [0.14–0.52; P < 0.01; k = 2]N/A [k = 0]
 OtherN/A [k = 0]0.26 [−0.69–1.20; P < 0.01; k = 4]N/A [k = 0]
Route of administration
 Subgroup differences?P < 0.01P < 0.01P < 0.01
 CNS1.73 [1.42 −2.04; P < 0.01; k = 33]0.62 [0.45–0.78; P < 0.01; k = 103]0.16 [−0.15–0.47; P = 0.21; k = 2]
 Systemic1.13 [0.83–1.42; P < 0.01; k = 29]1.53 [1.34–1.72; P < 0.01; k = 116]1.05 [0.87–1.22; P < 0.01; k = 75]
 Both2.83 [2.55–3.12; P = 0.47; k = 3]1.70 [1.32–2.08; P < 0.01; k = 11]N/A [k = 0]
Viral vectorsASOsSmall molecules
Total effect on survival per treatment type1.51 [1.28–1.74; P < 0.01]1.12 [0.99–1.26; P < 0.01]1.02 [0.85–1.20; P < 0.01]
Timing of treatment
 Subgroup differences?P < 0.01P < 0.01P = 0.07
 E2.13 [1.79–2.47; P = 0.28; k = 2]0.01 [−0.23–0.25; P = 0.46; k = 2]1.68 [0.35–3.01; P < 0.01; k = 3]
 P0/11.62 [1.35–1.88; P < 0.01; k = 49]1.20 [1.04–1.35; P < 0.01; k = 182]1.15 [−1.11–3.41; P < 0.01; k = 33]
 P21.21 [0.46–1.96; P < 0.01; k = 6]0.90 [0.53–1.26; P < 0.01; k = 33]0.74 [0.55–0.92; P < 0.01; k = 22]
 P3+0.92 [0.37–1.47; P < 0.01; k = 8]0.92 [0.57–1.27; P < 0.01; k = 13]1.10 [0.87–1.33; P < 0.01; k = 50]
Frequency of Administration
 Subgroup differences?P = 0.18P < 0.01N/A
 One dose1.47 [1.24–1.71; P < 0.01; k = 61]0.80 [0.64–0.97; P < 0.01; k = 120]N/A [k = 0]
 Multiple doses2.13 [1.20–3.06; P < 0.01; k = 4]1.48 [1.28–1.68; P < 0.01; k = 110]1.02 [0.85–1.20; P < 0.01; k = 77]
SMA model
 Subgroup differences?P < 0.01P < 0.01P < 0.01
 Severe Type I0.50 [−0.24–1.24; P = 0.03; k = 2]1.37 [0.97–1.77; P < 0.01; k = 39]0.00 [−0.36–0.36; N/A; k = 1]
 SMNΔ71.62 [1.36–1.88; P < 0.01; k = 53]0.75 [0.59–0.90; P < 0.01; k = 96]1.06 [0.85–1.28; P < 0.01; k = 58]
 TaiwaneseN/A [k = 0]1.51 [1.29–1.72; P < 0.01; k = 89]1.07 [0.68–1.46; P < 0.01; k = 9]
 Smn2B1.12 [0.73–1.51; P < 0.01; k = 10]N/A [k = 0]0.84 [0.36–1.32; P < 0.01; k = 9]
 BurgheronN/A [k = 0]0.33 [0.14–0.52; P < 0.01; k = 2]N/A [k = 0]
 OtherN/A [k = 0]0.26 [−0.69–1.20; P < 0.01; k = 4]N/A [k = 0]
Route of administration
 Subgroup differences?P < 0.01P < 0.01P < 0.01
 CNS1.73 [1.42 −2.04; P < 0.01; k = 33]0.62 [0.45–0.78; P < 0.01; k = 103]0.16 [−0.15–0.47; P = 0.21; k = 2]
 Systemic1.13 [0.83–1.42; P < 0.01; k = 29]1.53 [1.34–1.72; P < 0.01; k = 116]1.05 [0.87–1.22; P < 0.01; k = 75]
 Both2.83 [2.55–3.12; P = 0.47; k = 3]1.70 [1.32–2.08; P < 0.01; k = 11]N/A [k = 0]

K, number of comparisons per group. Values indicate effect size [95% CI].

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