Table 3.
Multi-modal findings in relation to hypotheses H1 to H6
A. Longitudinal change from Visit 1 to Visit 2 | ||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Marker | Region | Sample size (n) | Difference (%) | Effect size (dz)a | p | |||||||||
Cho | Frontal Lobe | 15 | 4.84 | −0.86 | 0.02* | |||||||||
Parietal Lobe | 15 | 4.35 | −0.81 | 0.01* | ||||||||||
Temporal Lobe | 15 | 6.21 | −0.44 | 0.12 | ||||||||||
Occipital Lobe | 15 | 4.81 | −0.59 | 0.08 | ||||||||||
Cr | Frontal Lobe | 15 | 3.35 | −0.55 | 0.08 | |||||||||
Parietal Lobe | 15 | 2.52 | −0.41 | 0.10 | ||||||||||
Temporal Lobe | 15 | 6.21 | −0.68 | 0.02* | ||||||||||
Occipital Lobe | 15 | 2.36 | −0.40 | 0.15 | ||||||||||
aTSC | Global GM | 17 | 9.98 | 1.20 | <0.01* | |||||||||
Global WM | 17 | 3.77 | 0.50 | 0.11 | ||||||||||
B. Rate of change relationships from Visit 1 to Visit 2 | ||||||||||||||
Marker | Region | Outcome | Sample size (n) | r | p | |||||||||
Cho | Frontal Lobe | RPQ | 15 | 0.52 | 0.05 | |||||||||
Parietal Lobe | 15 | 0.68 | <0.01* | |||||||||||
Temporal Lobe | 15 | 0.50 | 0.06 | |||||||||||
Occipital Lobe | 15 | 0.75 | <0.01* | |||||||||||
Frontal Lobe | BTACT | 13 | −0.33 | 0.27 | ||||||||||
Parietal Lobe | 13 | −0.49 | 0.09 | |||||||||||
Temporal Lobe | 13 | −0.48 | 0.10 | |||||||||||
Occipital Lobe | 13 | −0.26 | 0.38 | |||||||||||
Cr | Frontal Lobe | RPQ | 15 | 0.55 | 0.03* | |||||||||
Parietal Lobe | 15 | 0.52 | 0.05 | |||||||||||
Temporal Lobe | 15 | 0.65 | <0.01* | |||||||||||
Occipital Lobe | 15 | 0.43 | 0.11 | |||||||||||
Frontal Lobe | BTACT | 13 | −0.46 | 0.11 | ||||||||||
Parietal Lobe | 13 | −0.36 | 0.23 | |||||||||||
Temporal Lobe | 13 | −0.45 | 0.12 | |||||||||||
Occipital Lobe | 13 | −0.30 | 0.32 | |||||||||||
aTSC | Global GM | RPQ | 17 | 0.45 | 0.07 | |||||||||
Global WM | 17 | 0.36 | 0.12 | |||||||||||
Global GM | BTACT | 15 | −0.60 | 0.02* | ||||||||||
Global WM | 15 | 0.10 | 0.74 | |||||||||||
T2 | Body CC | RPQ | 13 | −0.61 | 0.03* | |||||||||
FA | Corona radiata | RPQ | 17 | 0.79 | <0.01* | |||||||||
BTACT | 15 | −0.65 | 0.01* | |||||||||||
C. Group differences at Visit 2 | ||||||||||||||
Marker | Region | Sample size (n) | Difference (%) | Effect size (d)b | p | |||||||||
Cho | Frontal Lobe | 16 patients / 23 controls | 1.77 | −0.13 | 0.66 | |||||||||
Parietal Lobe | 0.85 | −0.06 | 0.97 | |||||||||||
Temporal Lobe | 1.79 | −0.11 | 0.92 | |||||||||||
Occipital Lobe | 0.83 | −0.05 | 0.88 | |||||||||||
Cr | Frontal Lobe | 1.50 | 0.20 | 0.66 | ||||||||||
Parietal Lobe | 2.62 | 0.29 | 0.50 | |||||||||||
Temporal Lobe | 1.85 | −0.15 | 0.56 | |||||||||||
Occipital Lobe | 0.96 | 0.12 | 0.80 | |||||||||||
aTSC | Global GM | 17 patients / 23 controls | 1.36 | −0.24 | 0.51 | |||||||||
Global WM | 0.67 | −0.08 | 0.77 | |||||||||||
D. Cross-sectional relationships at Visit 2 | ||||||||||||||
Marker | Region | Outcome | Sample size (n) | r | p | |||||||||
Cho | Frontal Lobe | RPQ | 16 | 0.41 | 0.11 | |||||||||
Parietal Lobe | 16 | 0.27 | 0.31 | |||||||||||
Temporal Lobe | 16 | 0.53 | 0.03* | |||||||||||
Occipital Lobe | 16 | 0.42 | 0.11 | |||||||||||
Frontal Lobe | BTACT | 16 | 0.14 | 0.59 | ||||||||||
Parietal Lobe | 16 | 0.11 | 0.68 | |||||||||||
Temporal Lobe | 16 | 0.17 | 0.52 | |||||||||||
Occipital Lobe | 16 | 0.04 | 0.90 | |||||||||||
Cr | Frontal Lobe | RPQ | 16 | 0.45 | 0.08 | |||||||||
Parietal Lobe | 16 | 0.02 | 0.94 | |||||||||||
Temporal Lobe | 16 | 0.56 | 0.03* | |||||||||||
Occipital Lobe | 16 | 0.09 | 0.75 | |||||||||||
Frontal Lobe | BTACT | 16 | −0.10 | 0.72 | ||||||||||
Parietal Lobe | 16 | −0.06 | 0.84 | |||||||||||
Temporal Lobe | 16 | 0.17 | 0.52 | |||||||||||
Occipital Lobe | 16 | 0.50 | 0.05 | |||||||||||
aTSC | Global GM | RPQ | 17 | −0.15 | 0.56 | |||||||||
Global WM | 17 | 0.42 | 0.10 | |||||||||||
Global GM | BTACT | 17 | −0.01 | 0.96 | ||||||||||
Global WM | 17 | 0.11 | 0.68 | |||||||||||
T1 | Corona radiata | RPQ | 16 | 0.70 | <0.01* | |||||||||
Body CC | 16 | 0.51 | 0.04* | |||||||||||
Genu CC | 16 | 0.66 | <0.01* | |||||||||||
Splenium CC | 16 | 0.58 | 0.02* | |||||||||||
Frontal WM | 16 | 0.56 | 0.03* | |||||||||||
T2 | Genu CC | RPQ | 16 | 0.58 | 0.02* | |||||||||
ADC | Corona radiata | RPQ | 17 | 0.64 | <0.01* | |||||||||
FA | Frontal WM | RPQ | 17 | −0.56 | 0.02* | |||||||||
E. Predictive relationships with Visit 2 symptomatic and cognitive outcomes | ||||||||||||||
Marker | Region | Outcome | Sample size (n) | r | p | |||||||||
Cho | Frontal Lobe | RPQ | 18 | 0.18 | 0.48 | |||||||||
Parietal Lobe | 18 | 0.23 | 0.37 | |||||||||||
Temporal Lobe | 18 | 0.29 | 0.24 | |||||||||||
Occipital Lobe | 18 | 0.22 | 0.38 | |||||||||||
Frontal Lobe | BTACT | 18 | 0.08 | 0.75 | ||||||||||
Parietal Lobe | 18 | 0.11 | 0.65 | |||||||||||
Temporal Lobe | 18 | 0.30 | 0.23 | |||||||||||
Occipital Lobe | 18 | −0.02 | 0.93 | |||||||||||
Cr | Frontal Lobe | RPQ | 18 | −0.16 | 0.51 | |||||||||
Parietal Lobe | 18 | 0.20 | 0.43 | |||||||||||
Temporal Lobe | 18 | 0.24 | 0.35 | |||||||||||
Occipital Lobe | 18 | −0.04 | 0.87 | |||||||||||
Frontal Lobe | BTACT | 18 | 0.32 | 0.20 | ||||||||||
Parietal Lobe | 18 | 0.30 | 0.23 | |||||||||||
Temporal Lobe | 18 | 0.25 | 0.32 | |||||||||||
Occipital Lobe | 18 | 0.32 | 0.19 | |||||||||||
aTSC | Global GM | RPQ | 19 | −0.13 | 0.60 | |||||||||
Global WM | 19 | 0.02 | 0.94 | |||||||||||
Global GM | BTACT | 19 | 0.39 | 0.10 | ||||||||||
Global WM | 19 | 0.15 | 0.54 | |||||||||||
T1 | Corona radiata | BTACT | 15 | 0.58 | 0.02* | |||||||||
Body CC | 15 | 0.57 | 0.03* | |||||||||||
Genu CC | 15 | 0.82 | <0.01* | |||||||||||
Splenium CC | 15 | 0.57 | 0.03* | |||||||||||
Global WM | 15 | 0.58 | 0.03** | |||||||||||
ADC | Corona radiata | RPQ | 19 | 0.51 | 0.02* | |||||||||
Splenium CC | BTACT | 19 | 0.48 | 0.04* | ||||||||||
FA | Frontal WM | RPQ | 19 | −0.53 | 0.02* | |||||||||
F. Predictive relationships with Visit 3 symptomatic and cognitive outcomes | ||||||||||||||
Marker | Region | Outcome | Sample size (n) | r | p | |||||||||
Cho | Frontal Lobe | RPQ | 14 | 0.31 | 0.28 | |||||||||
Parietal Lobe | 14 | 0.36 | 0.21 | |||||||||||
Temporal Lobe | 14 | 0.24 | 0.41 | |||||||||||
Occipital Lobe | 14 | 0.23 | 0.43 | |||||||||||
Frontal Lobe | BTACT | 12 | 0.59 | 0.04* | ||||||||||
Parietal Lobe | 12 | 0.56 | 0.06 | |||||||||||
Temporal Lobe | 12 | 0.76 | <0.01* | |||||||||||
Occipital Lobe | 12 | 0.61 | 0.04* | |||||||||||
Cr | Frontal Lobe | RPQ | 14 | −0.17 | 0.55 | |||||||||
Parietal Lobe | 14 | 0.11 | 0.71 | |||||||||||
Temporal Lobe | 14 | 0.20 | 0.50 | |||||||||||
Occipital Lobe | 14 | 0.13 | 0.66 | |||||||||||
Frontal Lobe | BTACT | 12 | 0.45 | 0.14 | ||||||||||
Parietal Lobe | 12 | 0.46 | 0.13 | |||||||||||
Temporal Lobe | 12 | 0.57 | 0.05 | |||||||||||
Occipital Lobe | 12 | 0.85 | <0.01* | |||||||||||
aTSC | Global GM | RPQ | 15 | −0.30 | 0.29 | |||||||||
Global WM | 15 | 0.01 | 0.98 | |||||||||||
Global GM | BTACT | 13 | 0.19 | 0.52 | ||||||||||
Global WM | 13 | 0.61 | 0.03* | |||||||||||
T1 | Genu CC | BTACT | 10 | 0.71 | 0.03* | |||||||||
ADC | Genu CC | BTACT | 13 | 0.63 | 0.02* | |||||||||
Splenium CC | 13 | 0.64 | 0.02* | |||||||||||
G. Predictive relationships with Visit 2 functional outcome | ||||||||||||||
Marker | Region | Estimate | SE | Odds Ratio (95% Confidence Interval) | p | AUC | ||||||||
Cho | Frontal Lobe | −1.14 | 0.74 | 0.32 (0.08, 1.34) | 0.12 | 0.65 | ||||||||
Parietal Lobe | −1.51 | 0.85 | 0.22 (0.04, 1.17) | 0.08 | 0.72 | |||||||||
Temporal Lobe | −0.84 | 0.69 | 0.43 (0.11, 1.68) | 0.22 | 0.67 | |||||||||
Occipital Lobe | −0.07 | 0.61 | 0.94 (0.28, 3.10) | 0.91 | 0.54 | |||||||||
Cr | Frontal Lobe | −0.20 | 0.34 | 0.82 (0.43, 1.58) | 0.55 | 0.59 | ||||||||
Parietal Lobe | −0.16 | 0.31 | 0.85 (0.46, 1.58) | 0.61 | 0.58 | |||||||||
Temporal Lobe | 0.12 | 0.25 | 1.13 (0.69, 1.86) | 0.63 | 0.53 | |||||||||
Occipital Lobe | −0.04 | 0.27 | 0.96 (0.57, 1.64) | 0.89 | 0.55 | |||||||||
aTSC | Global GM | −0.13 | 0.22 | 0.88 (0.58, 1.34) | 0.56 | 0.56 | ||||||||
Global WM | 0.11 | 0.15 | 1.12 (0.83, 1.51) | 0.46 | 0.54 |
dz < 0 denotes decrease (Visit 1 to Visit 2)
d < 0, denotes lower values in patients vs. controls
CC = corpus callosum; SE = standard error; AUC = area under the curve
Note. Markers were predominately measured within WM with the exception of aTSC. For brevity, only the statistically significant (*p < 0.05) findings involving 23Na MRI, MRF, and DTI markers are presented (see Table S3 for overlap with prior publications and Table S6 for NAA and mI). (A) The Wilcoxon signed-rank test (WSRT) was used to evaluate whether markers from Visit 1 were significantly different from those at Visit 2, following H1. (B) Spearman correlations were used to evaluate whether rates of change in markers were associated with rates of change in symptomatic and cognitive outcomes, from Visit 1 to Visit 2, following H2. (C) The Mann-Whitney U (MWU) test was used to evaluate whether markers from Visit 2 were significantly different in patients compared to controls, following H3. (D) Spearman correlations were used to evaluate whether markers at Visit 2 cross-sectionally correlated with symptomatic and cognitive outcomes at Visit 2, following H4. (E) Spearman correlations were used to evaluate whether markers at Visit 1 serially correlated with symptomatic and cognitive outcomes at Visit 2 or (F) Visit 3, following H5. (G) Logistic regression was used to evaluate whether markers at Visit 1 were significantly associated with functional outcome at Visit 2, following H6. Additionally, receiver operating characteristic curve analysis assessed the overall discriminative ability of the logistic regression models for predicting functional outcome at Visit 2.