A conceptual disease model for adult Pompe disease

Table 2 Regression coefficients based on a random effects model

Level	Physiological variables		Symptom status	Functional status	Health perceptions	Health related quality of life
Model	I MRC	II FVC	III FSS	IV RHS	V EQ-5D VAS	VI MCS	VII PCS	VIII Utility
Constant	83.452	70.526	8.737	16.989	−9.313	49.990	24.526	0.530
Age	−0.069	−0.144	0.011	−0.096	0.126	−0.016	−0.046	0.001
Female	−0.028	17.994	0.307	−0.957	4.673	−1.282	−0.402	0.010
Disease duration	−0.563	−0.346	−0.077	−0.052	0.653	0.063	−0.086	−0.001
Enzyme activity	0.370	−0.431	0.007	−0.048	0.004	−0.359	−0.069	−0.006
Muscle strength			−0.031	0.189
Respiratory function			−0.011	0.075
Fatigue Severity Scale				−0.578
Rotterdam Handicap Scale					2.030
Visual Analogue Scale						0.157	0.223	0.004
patients	78	72	61	61	73	73	73	71
observations	249	257	160	158	154	152	152	161
R² (overall)	0.105	0.075	0.322	0.623	0.343	0.183	0.293	0.354
Wald test (p-value)	<0.001	0.001	0.002	<0.001	<0.001	0.005	<0.001	<0.001

Figures in bold indicate significance at 5 % level; Enzyme activity, muscle strength and respiratory function were measured as a percentage of normal values, Visual Analogue Scale, Mental Component Scale and Physical Component Scale were measured on a scale from 0–100, Fatigue Severity Scale was measured on a scale from 1–7, Rotterdam Handicap Scale was measured on a scale from 0–36, Utility was measured on a scale from 0–1

ISSN: 1750-1172