Higher dose alglucosidase alfa is associated with improved overall survival in infantile-onset Pompe disease (IOPD): data from the Pompe Registry

Table 4 Relative risk of death according to average alglucosidase alfa dose by age and CRIM status

	N patients	Person-years	N deaths	Adjusted HR^a	95% CI	p-value
Stratified by age at first treatment (p-value for interaction^b)	(0.8104)
Age at first treatment < 3 months: Per 1-unit increase in average dose^c	144	706	22	0.29	0.09, 0.90	0.0326
Age at first treatment ≥ 3 months: Per 1-unit increase in average dose^c	188	726	66	0.43	0.21, 0.87	0.0195
Stratified by CRIM status (p-value for interaction^b)	(0.7051)
CRIM-positive: Per 1-unit increase in average dose^c	213	1142	41	0.44	0.20, 0.98	0.0438
CRIM-negative: Per 1-unit increase in average dose^c	70	289	29	0.24	0.04, 1.37	0.1088

CRIM, cross-reactive immunological material
^aModels are adjusted for age (as time scale), age at first treatment, sex, and CRIM/ITI group
^bp-value for interaction between the stratification variable and average dose over time, i.e., is the association between average dose and survival significantly different for the two subgroups. Calculated from likelihood ratio test comparing the main (non-stratified) model to the main model with an interaction term
^cAverage dose received over time from first treatment, updated over time, measured in multiples of the label dose. Range: > 0 to 4 times label dose

ISSN: 1750-1172