Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational study

Table 3 Risk of death for 283 adults with Pompe disease applying time-dependent cox regression*

	Model 1a†
Time-dependent cox regression model	HR	95% CI	P Value
ERT‡	0.41	0.19 to 0.87	0.02
Age (in quartiles)			0.14
<37 years (ref)	1
37-48 years	1.26	0.38 to 4.12	0.71
48-57 years	1.42	0.44 to 4.61	0.56
≥57 years	2.57	0.86 to 7.72	0.09
Sex	1.01	0.55 to 1.87	0.98
Disease severity			0.001
No wheelchair use or respiratory support ‡	1
Wheelchair use	2.87	0.98 to 8.36	0.05
Use of respiratory support	2.05	0.62 to 6.77	0.24
Both wheelchair use and respiratory support	5.32	2.25 to 12.56	<0.001
Country of residence			0.13
Netherlands (ref)	1
United Kingdom	1.35	0.46 to 3.95	0.58
United States	2.14	1.01 to 4.55	0.05
Germany	0.62	0.19 to 1.95	0.41
Other§	1.23	0.42 to 3.63	0.70

* ERT denotes enzyme replacement therapy and HR hazard ratio.
† Intent-to-treat approach with ERT, age categories, and disease severity as time-dependent covariates.
‡ Values for ERT were derived after adjustment for age, sex, disease severity, and country of residence.
§ Including patients from Australia and Canada.
‡Respiratory support includes partial and full-time invasive and non-invasive respiratory support.

ISSN: 1750-1172