Sleep disordered breathing in mucopolysaccharidosis I: a multivariate analysis of patient, therapeutic and metabolic correlators modifying long term clinical outcome

Table 3 Correlators of sleep related clinical outcome based on multivariate analysis

Outcome	Modifier	Patient group	Exp (β)	Lower 95% CI	Upper 95% CI	p-value
SDB Present	Phenotype	All (n = 61)				ns
	Treatment					ns
	Age @ start of treatment					ns
	Follow-up duration					ns
	DS:CS ratio @ 1 year		−5.94	−12.39	−1.61	0.0017
Mean ODI 4%	Phenotype	All (n = 61)				ns
	Treatment					ns
	Age @ start of treatment					ns
	Follow-up duration					ns
	DS:CS ratio @ 1 year		6.35	1.77	10.95	0.008
Therapeutic Intervention	Phenotype	All (n = 61)				ns
	Treatment					ns
	Age @ start of treatment		−0.21	−0.45	−0.03	0.012
	Follow-up duration					ns
	DS:CS ratio @ 1 year					ns
SDB Present	Donor Type	HSCT Treated Hurler (n = 41)				ns
	IDUA level @ 1 year		0.08	0.02	0.16	0.004
	Age @ start of treatment					ns
	Follow-up duration					ns
	DS:CS ratio @ 1 year					ns
ODI 4% at time of antibody status	DS:CS Ratio	ERT Treated Attenuated (n = 17)				ns
	Inhibitory Antibodies		−8.27	−11.19	−5.36	0.001
	Age @ start of treatment					ns
	Follow-up duration					ns

p-values for modifiers based on multivariate analysis using stepwise fit (minimum AICc) followed by standard least squares for continuous outcomes and logistic fit for binomial outcomes. CI indicates confidence interval; ERT, enzyme replacement therapy; HSCT, haemopoietic stem cell transplant; IDUA, alpha-L-iduronidase enzyme level; SDB, Sleep disordered breathing; ODI 4%, oxygen desaturation index 4%. Inhibitory antibodies defined as IgG titre > 4000 and uptake inhibition >30%.

ISSN: 1750-1172