Long-term survival with sebelipase alfa enzyme replacement therapy in infants with rapidly progressive lysosomal acid lipase deficiency: final results from 2 open-label studies

Table 4 Sebelipase alfa dosing status and immunogenicity

	VITAL (N = 9)	CL08 (N = 10)
Final dose, surviving patients, n/N	5/9	8/10
1.0 mg/kg qw, n	0	1^a
3.0 mg/kg qw, n	3	3
5.0 mg/kg qw, n	2	4
Week of first 3.0 mg/kg dose, median (range)^b	14 (6–91)	12 (4–58)
Immunogenicity during the study^c
Anti-drug antibody test, n positive/N tested	4/7	6/10
Neutralizing assay, n positive/N tested	3/4	6/6
Inhibit cellular uptake of LAL	3/4	6/6
Inhibit LAL enzyme activity	2/4^d	6/6

LAL lysosomal acid lipase, qw once weekly
^aOne patient received successive dose escalations up to a maximum dose of 7.5 mg/kg qw. This patient had subsequent successive dose reductions to 1.0 mg/kg qw after a successfully engrafted bone marrow transplant at week 101
^bAmong surviving patients
^cAmong all patients with posttreatment assessments
^dThese patients also had neutralizing antibodies that inhibit cellular uptake of LAL

ISSN: 1750-1172