Article Figures & Data
Figures
- Fig 1.
Visualization of the fit of the Weibull distribution on cumulative enhancing volume data of patients with enhancing-lesion volumes in dataset A, by means of a probability attenuation function (attenuation on the y-axis describes the probability for enhancing-lesion volume to occur at a given point).
Tables
Characteristics Dataset A (n = 169) Dataset B, Placebo (n = 69) Patient Female/male 123:46 50:19 RRMS/SPMS with relapses 169:0 57:12 Mean age (SD) 35.4 (8.4) 38.5 (9.1) Mean disease duration in years (SD) 6.4 (5.3) 5.9 (5.9) Median baseline EDSS (IQR) 2.0 (1.5–3.5) 2.5 (1.5–3.5) MR imaging % Inactive patients 41.4% 69.6% Mean number of enhancing lesions (SD) 2.4 (4.1) 1.3 (0.4) -
Note:—EDSS indicates Expanded Disability Status Scale; IQR, interquartile range.
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Study No.b FU Time (mo) % Inactive Patients (Placebo) Efficacy Measure Absolute % Increase in Inactive Patients Lesion No. (All Patients) Lesion Volume (All Patients) Lesion Volume (Active Patients) Oral temsirolimus 120 9 25% +2% −26% −29% −26% Glatiramer acetate 239 9 5% −2% −33%c −42% −43% Natalizumab 3 mg 139 6 32% +43% −88% −87% −64% 6 mg 145 6 32% +33% −82% −76% −54% Oral fingolimod 1.25 mg 164 6 47% +30% −43% −50% +16% 5 mg 158 6 47% +35% −61% −63% +10% Overall mean effect 13% 56% 58% 27% -
Note:—FU indicates follow-up.
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↵a Effects shown are the percentage increase in active patients, the percentage change in mean or median number of lesions, the percentage change in mean lesion volume in the complete cohort, and the percentage change in mean lesion volume in active patients.
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↵b Total number of participating patients in trial.
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↵c Median lesion number.
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Distributions Anderson-Darling Kolmogorov-Smirnov Dataset A Dataset B Dataset A Dataset B Weibull >.2 >.2 .77 .77 γ >.2 .1 < P < .2 .88 .12 Log-normal 0.1 < P < .2 >.2 .06 .99 Normal (cube root transformation) >.2 >.2 .60 .36 Normal (Natural logarithm transformation) 0.1 < P < .2 >.2 .06 .99 Normal (Log10 transformation) 0.1 < P < .2 >.2 .06 .99 Normal <.01 <.01 <.01 <.01 Inverse Gaussian <.01 <.05 <.01 .49 -
↵a Results of the Anderson-Darling and Kolmogorov-Smirnov goodness-of-fit test for the cumulative volume of enhancing lesions in active patients in datasets A (n = 133) and B (n = 44). Data are P values.
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% Inactive Patients in Placebo Group % Decrease in Mean Lesion Volume of Active Patients Absolute % Increase in Inactive Patientsb 0% 10% 20% 30% 40% 50% 10 0 – – – 435 225 124 10 518 309 204 139 86 65 20 128 101 82 65 47 38 30 56 50 43 35 30 27 40 31 28 24 22 21 19 50 20 19 18 17 16 16 40 0 – – – – – 580 10 470 369 315 251 199 153 20 121 115 94 83 75 66 30 49 44 44 40 37 36 40 25 24 23 24 21 20 50 15 15 15 15 14 15 -
↵a Number of patients per treatment arm necessary to perform parallel-group-designed trials with a statistical power of 80%, to detect treatment effects ranging from 0% to 50% increase in inactive patients and a 0%–50% reduction in mean enhancing-lesion volume in active patients.
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↵b – Indicates percentage increase of patients developing no/zero enhancing lesions during follow-up.
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- Table 5:
Number of patients per treatment arm necessary to perform parallel grouped placebo-controlled clinical trialsa
% Decrease in Mean Number of Enhancing Lesions No. 50 129 60 80 70 47 80 28 90 12 -
↵a To detect a percentage decrease in the mean number of enhancing lesions ranging from 50% to 90%, for a power of 80%.
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