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Table 2 Ordered Logistic Regression Results for Annual Health-State Transitions in Gaucher Disease Type 1

From: A new framework for evaluating the health impacts of treatment for Gaucher disease type 1

  Proportion for estimation sample Coefficient Robust standard error 95% CI
Health State in Previous Year (vs. Mild)
 Mild + Bone Pain 0.114 1.305*** 0.241 0.833, 1.777
 Mild + SSC 0.010 0.840* 0.452 −0.046, 1.727
 Moderate 0.344 2.581*** 0.305 1.983, 3.179
 Moderate + SSC 0.041 1.253*** 0.381 0.506, 2.000
 Marked 0.028 4.504*** 0.465 3.593, 5.416
 Marked + SSC 0.062 3.617*** 0.462 2.711, 4.524
 Severe 0.003 4.213*** 0.698 2.845, 5.581
 Severe + SSC 0.007 6.072*** 1.374 3.380, 8.765
Years on Treatment (vs. One Year)
 Two Years 0.086 0.293 0.361 −0.414, 000
 Three or More Years 0.807 0.315 0.305 −0.283, 0.913
Baseline DS3 Category (vs. Mild)
 Moderate 0.312 −0.150 0.276 −0.691, 0.391
 Marked 0.410 0.873*** 0.274 0.336, 1.410
 Severe 0.071 1.349*** 0.411 0.543, 2.154
Not Splenectomized 0.672 −1.089*** 0.250 −1.578, −0.599
Ordered Logistic Cutpoints
 Cutpoint 1   0.740 0.456 −0.153, 1.634
 Cutpoint 2   1.662*** 0.464 0.752, 2.572
 Cutpoint 3   1.727*** 0.467 0.812, 2.641
 Cutpoint 4   5.054*** 0.519 4.037, 6.070
 Cutpoint 5   5.835*** 0.523 4.810, 6.861
 Cutpoint 6   6.577*** 0.633 5.335, 7.818
 Cutpoint 7   8.808*** 0.639 7.555, 10.060
 Cutpoint 8   9.066*** 0.650 7.792, 10.340
Number of Patients 130    
Number of Observations 970    
  1. Standard errors account for multiple observations per patient. *** p < 0.01, **p < 0.05, *p < 0.10
  2. Abbreviations: CI confidence interval, SSC severe skeletal complications