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Table 4 Sebelipase alfa dosing status and immunogenicity

From: 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

  VITAL (N = 9) CL08 (N = 10)
Final dose, surviving patients, n/N 5/9 8/10
 1.0 mg/kg qw, n 0 1a
 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 studyc   
 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 2/4d 6/6
  1. LAL lysosomal acid lipase, qw once weekly
  2. 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
  3. bAmong surviving patients
  4. cAmong all patients with posttreatment assessments
  5. dThese patients also had neutralizing antibodies that inhibit cellular uptake of LAL