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

Table 3 Relative risk of death or invasive ventilation according to average relative dose of alglucosidase alfa

	Person Years	N Deaths	Adjusted HR^a	95% CI	p-value
Outcome: Risk of death
Average relative dose over time, continuous (range: > 0 to 4.0 times label dose)^b
Per 1-unit increase in average dose	1609	88	0.40	0.22, 0.73	0.0030
Categories of average dose over time^b
Below label dose	23	8	7.16	2.28, 22.48	0.0008
Label dose	854	52	1.00 (reference)	–	–
Between label and double dose	339	14	0.87	0.41, 1.82	0.7020
Double dose	249	12	0.83	0.38, 1.82	0.6340
Above double to quadruple dose	144	2	0.10	0.01, 0.82	0.0312
Outcome: Risk of death or invasive ventilation
Average relative dose over time, continuous (range: > 0 to 4.0 times label dose)^b
Per 1-unit increase in average dose	1237	97	0.48	0.28, 0.84	0.0100
Categories of average relative dose over time^b
Below label dose	21	5	1.59	0.44, 5.73	0.4810
Label dose	633	60	1.00 (reference)	–	–
Between label and double dose	284	16	0.70	0.34, 1.46	0.3405
Double dose	189	16	0.96	0.47, 2.00	0.9224
Above double to quadruple dose	110	0	0.00	NE, NE	0.9895

CRIM, cross-reactive immunological material; EOW, every other week; ITI, immune tolerance induction; NE, not evaluable
^aModels are adjusted for age (as time scale), age at first treatment, sex, and CRIM/ITI group
^bAverage dose received over time from first treatment, updated over time, measured in multiples of the label dose, ranging from > 0 to 4 times label dose. Average dose categories: 'Below label dose' is average dose < 0.95;'Label dose' is from 0.95 to < 1.05; 'Between label and double dose' is from 1.05 to < 1.75; 'Double dose' is 1.75 to < 2.25; 'Above double to quadruple dose' is 2.25 to 4.0

ISSN: 1750-1172