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

Table 2 Ordered Logistic Regression Results for Annual Health-State Transitions in 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

Standard errors account for multiple observations per patient. *** p < 0.01, **p < 0.05, *p < 0.10
Abbreviations: CI confidence interval, SSC severe skeletal complications

ISSN: 1750-1172