Enzyme replacement therapy reduces the risk for wheelchair dependency in adult Pompe patients

Table 2 Multivariate time-dependent Cox regression analysis of wheelchair use (A) and the need for respiratory support (B)

	Events	Person-years	HR	95% CI	p-value
A. Wheelchair use
Treatment^a
Untreated (ref)	30	468
ERT	16	652	0.36	(0.17–0.75)	0.004
Disease duration^a
< 5 years (ref)	10	464
5–10 years	10	332	1.19	(0.41–3.48)	1
> 10 years	26	324	3.87	(1.55–9.61)	0.002
Sex
Male (ref)	17	583
Female	29	537	1.80	(0.88–3.69)	0.13
Country of residence
Netherlands (ref)	13	531
Germany	9	173	1.19	(0.41–3.47)	1
US	12	214	1.95	(0.73–5.22)	0.13
Other	12	202	2.99	(1.17–7.67)	0.018
B. Respiratory support
Treatment^a
Non-use (ref)	20	529
ERT	28	661	1.23	(0.61–2.47)	0.51
Disease duration^a
< 5 years (ref)	11	430
5–10 years	12	319	1.12	(0.42–3.02)	1
> 10 years	25	441	2.13	(0.89–5.09)	0.051
Sex
Male (ref)	21	420
Female	27	770	0.67	(0.34–1.32)	0.18
Country of residence
Netherlands (ref)	18	599
Germany	9	189	1.28	(0.48–3.38)	1
US	13	213	2.22	(0.90–5.49)	0.10
Other	8	189	1.37	(0.52–3.62)	0.46

^aTime-dependent covariates, updated at start of ERT; Italicized p-values are significant (i.e. below 0.05).

ISSN: 1750-1172