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Table 2 Parameter estimation of the final mode of OS

From: Accelerating drug development for amyotrophic lateral sclerosis: construction and application of a disease course model using historical placebo group data

 

Estimate

RSE (%)

Bootstrap (983)*

Median (95% CI)

SIGM

0.916

5.80

0.920 (0.820–1.03)

MU

3.04

2.80

3.06 (2.87–3.26)

θDuration

0.0317

28.3

0.0311 (0.0109–0.0511)

θRiluzole

− 0.228

57.5

− 0.217 (− 0.508–0.101)

η (SIGM), %

25.2

15.6

23.8 (16.2–31.5)

η (MU), %

7.20

19.4

6.70 (4.00–10.1)

εadd

0.693

17.2

0.686 (0.503–0.918)

  1. SIGM the median of the lognormal distribution, MU the variance of the lognormal distribution, θDuration is the covariant parameter of the disease duration, θRiluzole is the is the covariant parameter of the proportion of patients treated with Riluzole, η is the inter-study variability of model parameter, εadd additive residual error; RSE, relative standard error, CI confidience interval
  2. *983 means that the model succeeded 983 times out of 1000 bootstrap attempt
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172