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Table 4 mPRO and QoL scale results across the 6-month period with overall cohort averages and statistical significance

From: A feasibility study of mHealth and wearable technology in late onset GM2 gangliosidosis (Tay-Sachs and Sandhoff Disease)

Mean (Median)
[min-max]
M0 M1 M2 M3 M4 M5 M6 Overall Cohort Wilcoxon p-value Hollander p-value Reference range
Tremor impact 0.0 (0.0)
[0.00–0.00]
0.4 (0.0)
[0.00–1.00]
0.8 (0.5)
[0.00–2.00]
1.0 (1.0) [0.00–2.00] 0.7 (0.5)
[0.00–2.00]
0.9 (1.0)
[0.00–2.00]
1.3 (1.0)
[0.00–2.00]
0.7 (0.5)
[0.00–2.00]
0.05 0.9375 0–3
Higher score: more tremor
Disease Impact 4.5 (5.0)
[2.00–7.00]
4.9 (4.5)
[0.00–10.00]
4.1 (4.0)
[1.00–6.00]
5.8 (6.5) [1.00–10.00] 5.6 (5.0)
[2.00–9.00]
6.1 (5.0)
[3.00–10.00]
5.7 (5.5)
[3.00–9.00]
5.2 (5.0)
[0.00–10.00]
0.20 0 0–19
Higher score = higher impact
Impact on the family 6 (6.0)
[2.00–9.00]
6.1 (6.0)
[4.00–9.00]
5.6 (6.0)
[2.00–8.00]
6.3 (6.0) [5.00–8.00] 6.3 (6.0)
[5.00–8.00]
6.1 (6.0)
[5.00–8.00]
6.3 (6.5)
[5.00–8.00]
6.1 (6.0)
[2.00–9.00]
0.79 0 0–12
Higher score = higher impact
Wider impact 1.3 (0.0)
[0.00–7.00]
1.3 (0.0)
[0.00–7.00]
2.6 (1.0)
[0.00–7.00]
1.2 (0.0) [0.00–6.00] 1.6 (0.0)
[0.00–7.00]
0.9 (0.0)
[0.00–5.00]
1.8 (1.0)
[0.00–7.00]
1.5 (0.0)
[0.00–7.00]
1.0 0 0–14
Higher score = higher impact
Impact Composite Scale 0.27 (0.28)
[0.11–0.40]
0.28 (0.31)
[0.10–0.40]
0.29 (0.27)
[0.08–0.44]
0.32 (0.34) [0.15–0.45] 0.27 (0.25)
[0.00–0.49]
0.30 (0.29)
[0.21–0.40]
0.31 (0.31)
[0.23–0.40]
0.27 (0.29)
[0.00–0.49]
0.42 0 0–1
Higher score = higher impact
Perceived Stress 17.9 (18.5)
[11.00–26.00]
NA 19.6 (21.0)
[7.00–25.00]
NA 16.3 (18.0)
[9.00–24.00]
NA 20.0 (20)
[12.00–29.00]
18.4 (19.3)
[7.00–29.00]
0.04 0.8742 0–40
Higher score = higher stress
Global Self Worth 17.0 (17.5)
[12.00–24.00]
NA 16.0 (15.5)
[13.00–22.00]
NA 17.8 (18.0)
[14.00–22.00]
NA 18.1 (18.0)
[14.00–24.00]
17.2 (17.8)
[12.00–24.00]
0.59 0.5 0–24
Higher score = higher self-worth
Rosenberg Self Esteem 15.0 (15.0)
[11.00–17.00]
NA 15.3 (15.5)
[11.00–19.00]
NA 14.4 (14.0)
[13.00–16.00]
NA 15.4 (15.0)
[13.00–19.00]
15.0 (15.0)
[11.00–19.00]
0.68 0 0–30
Higher score = higher self esteem
CHU 9D 0.87 (0.88)
[0.67–0.96]
NA 0.84 (0.83)
[0.66–1.00]
NA 0.83 (0.83)
[0.68–0.92]
NA 0.83 (0.84)
[0.72–0.95]
0.84 (0.83)
[0.66–1.00]
0.36 0.438 0.33–1
Higher score = better health
PedsQL Multi-dimensional Fatigue 49.5 (46.5)
[31.94–73.61]
46.5 (43.1)
[27.78–75.00]
46.7 (43.1)
[38.89–66.67]
49.7 (45.8) [34.72–73.61] 46.6 (45.1)
[29.17–75.00]
50.2 (50.0)
[31.94–83.33]
61.1 (58.3)
[50.00–75.00]
49.6 (45.8)
[27.78–83.33]
0.73 0 0–100
Higher score = less fatigue
  1. CHU 9D Child Health Utility 9D, M month