Analysis of fatal outcomes of patients with mucopolysaccharidosis type II according to the Russian mucopolysaccharidosis registry

Table 4 The main predictors of the fatal outcomes (univariant analysis) in patients with mucopolysaccharidosis type II

MPS type II feature	Sensitivity	Specificity	OR (95%CI)	P value
Diagnosis age > 4 years	70.0	72.8	6.3 (2.2; 17.6)	0.0002
ERT age > 5 years	87.5	59.4	10.2 (2.2; 47.5)	0.0005
Birth weight > 3.88 kg	62.5	72.3	4.4 (0.96; 19.7)	0.042
Age > 13 years	75.0	65.0	5.6 (1.9; 16.2)	0.0006
Hydrocephalus	73.3	58.6	3.9 (1.2; 13.0)	0.019
Epilepsia	53.3	89.9	10.2 (3.1; 33.1)	0.000009
Swallowing disturbances	68.8	77.9	7.4 (2.3; 23.1)	0.0002
Impossible to walk after 12 years	61.5	77.9	5.7 (1.6; 19.9)	0,002
Heart disease	100.0	23.3	-	0.031
Cardiomyopathy	60.0	70.7	3.6 (1.2; 11.0)	0.017
Myxomatous valve disease	100.0	32.3	-	0.009
Deafness	92.3	32.7	5.8 (0.7; 46.7)	0.062
Respiratory disorders	87.5	60.2	10.6 (2.3; 48.7)	0.001
Apnoe at the diagnosis	54.5	73.6	3.4 (0.92; 12.3)	0.057
Apnoe at the last visit	77.8	70.2	8.3 (1.6; 42.6)	0.004
Obstructive disease	72.7	66.3	5.2 (1.3; 21.2)	0.012
Hernias	100.0	16.9	-	0.059

ERT: Enzyme replacement therapy; MPS: Mucopolysaccharidosis; OR: Odds ratio.