The objective of this prospective, open-label, single-arm, Phase III study was to assess the efficacy and safety of Privigen ®, the first 10% liquid intravenous immunoglobulin stabilized with L-proline, in patients with primary immunodeficiency. As part of a larger study, 19 children (3-11 years) and 12 adolescents (12-15 years) received Privigen 200-741 mg/kg intravenously for 12 months at a maximum infusion rate of 8 mg/kg/min. Thirteen patients received infusions at up to 12 mg/kg/min as part of an extension study. The primary endpoint was the annual rate of acute serious bacterial infections (aSBIs) per patient. Privigen was effective, as indicated by the annual rate of aSBI of 0.12 per patient (upper 1-sided 99% CI 0.499) in 3- to 11-year-olds and 0.10 (CI 0.642) in 12- to 15-year-olds. Three patients experienced 1 aSBI each, but did not discontinue treatment. The annual rate of all infections (including aSBIs) per patient was 4.63 in children and 2.42 in adolescents. The annual rate of days missed from school/day care was 11.5 days in children and 4.8 days in adolescents, consistent with the low annual rate of hospitalization (0.53 days and 0 days, respectively). Privigen administration was well-tolerated even at high infusion rates. The proportion of infusions with temporally associated AEs for the entire pediatric population was 0.18 (upper 1-sided 95% CI 0.221). One patient experienced serious AEs considered related to study drug and was withdrawn. Privigen was effective in sustaining a low incidence of infections, including aSBIs, and showed a good tolerability profile, with adverse events typical of disease and treatment.
ASJC Scopus subject areas
- Pediatrics, Perinatology, and Child Health
- Immunology and Allergy
- Pulmonary and Respiratory Medicine