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Table 3 Mixed-effects models for repeated measuresa analyses of the changes of all outcome measures at each time point from the baseline (nusinersen start time)

From: Enhancing the effects of nusinersen with cybernic treatment using Hybrid Assistive Limb (HAL) in spinal muscular atrophy: a real-world case series and exploratory cohort analysis

 

Cohort 1 (Conventional therapy plus HAL)

Cohort 2 (Conventional therapy only)

Difference between cohorts

n

LSM

SEM

[95% CI for LSM]

n

LSM

SEM

[95% CI for LSM]

LSM

[95% CI for LSM]

p-value

HFMSE total score

           

 3 months (12 weeks)

5

1.7

1.06

[− 0.7, 4.1]

7

1.6

0.89

[− 0.4, 3.7]

0.1

[− 3.1, 3.3]

0.945

 9 months

5

1.7

2.27

[− 3.3, 6.8]

7

1.1

1.92

[− 3.2, 5.3]

0.7

[− 6.0, 7.3]

0.826

 15 months

5

4.7

1.13

[2.2, 7.3]

7

2.9

0.96

[0.7, 5.1]

1.8

[− 1.6, 5.2]

0.254

RULM total score

           

 3 months (12 weeks)

5

0.0

0.55

[− 1.2, 1.1]

5

0.0

0.55

[− 1.1, 1.2]

-0.1

[− 1.7, 1.6]

0.919

 9 months

5

0.6

0.55

[− 0.6, 1.7]

5

−0.2

0.55

[− 1.3, 1.0]

0.7

[− 0.9, 2.4]

0.376

 15 months

5

2.2

0.55

[1.0, 3.3]

4

−0.2

0.65

[− 1.5, 1.2]

2.3

[0.5, 4.2]

0.016

2MWT, m

           

 3 months (12 weeks)

5

28.19

12.19

[− 1.60, 57.98]

4

4.19

13.78

[− 29.48, 37.85]

24.01

[− 23.03, 71.04]

0.259

 9 months

5

32.23

12.33

[2.27, 62.19]

4

3.51

13.93

[− 30.34, 37.36]

28.71

[− 18.54, 75.97]

0.189

 15 months

5

34.57

12.28

[4.57, 64.57]

4

−3.86

13.88

[− 37.75, 30.03]

38.44

[− 8.88, 85.75]

0.094

10MWT

           

 Gait speed, m/sec

           

  3 months (12 weeks)

5

0.113

0.084

[− 0.090, 0.317]

4

0.071

0.094

[− 0.156, 0.298]

0.043

[− 0.262, 0.347]

0.747

  9 months

5

0.207

0.093

[− 0.015, 0.430]

4

0.116

0.104

[− 0.133, 0.365]

0.092

[− 0.242, 0.425]

0.535

  15 months

5

0.233

0.077

[0.048, 0.419]

4

0.138

0.086

[− 0.069, 0.346]

0.095

[− 0.183, 0.373]

0.44

 Step length, m

           

  3 months (12 weeks)

5

0.029

0.030

[− 0.045, 0.104]

4

0.011

0.034

[− 0.072, 0.094]

0.019

[− 0.093, 0.130]

0.697

  9 months

5

0.041

0.029

[− 0.029, 0.112]

4

0.021

0.032

[− 0.058, 0.099]

0.021

[− 0.085, 0.127]

0.647

  15 months

5

0.121

0.039

[0.026, 0.217]

4

0.036

0.043

[− 0.071, 0.143]

0.086

[-0.058, 0.229]

0.194

 Cadence, step/sec

           

  3 months (12 weeks)

5

0.136

0.105

[− 0.120, 0.392]

4

0.055

0.118

[− 0.235, 0.344]

0.081

[− 0.325, 0.487]

0.642

  9 months

5

0.320

0.148

[− 0.038, 0.678]

4

0.100

0.167

[− 0.302, 0.502]

0.220

[− 0.328, 0.768]

0.37

  15 months

5

0.190

0.114

[− 0.085, 0.466]

4

0.110

0.129

[− 0.201, 0.421]

0.080

[− 0.352, 0.512]

0.67

  1. 2MWT, 2-minute walk test; 10MWT, 10-rmetre walk test; CI, confidence interval; HAL, Hybrid Assistive Limb; HFMSE, Hammersmith Functional Motor Scale-Expanded; LSM, least squares mean; PT, physiotherapy; RULM, Revised Upper Limb Module; SEM, standard error of the mean
  2. aIn the analytical model, the group was defined as a fixed effect of the categorical variable. The analytical model included time point, group, and time point group interactions. Baseline measures were used as covariates as fixed effects for continuous variables. The Kenward-Roger method was used to estimate the degrees of freedom. The variance-covariance structure was assumed to be unstructured
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172