Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease

Table 1 Patient characteristics for patients in survival analyses

	Kishnani (2009)	Nicolino (2009)	Erasmus MC
N	18	21	20
Deaths	5 (28%)	6 (29%)	4 (20%)
Age at end study in months [range]	34.5 [19.7-44.0]	41.0 [7.7-80.3]	60.9 [3.2-178.8]
Patients using ventilation	9 (50%)	7 (33%)	5 (25%)^b
Dosage (every other week)
15 mg/kg	0	0	2^{c, d}
20 mg/kg	9	21^a	8^e
40 mg/kg	9	0	10^d

^aEight patients switched to 40 mg/kg after 26 weeks due to clinical deterioration; ^bTwo patients died; ^cBoth patients switched to 30 mg/kg and later to 40 mg/kg; ^dEvery week; ^eFive patients switched to 40 mg/kg.

ISSN: 1750-1172