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Table III Efficacy endpoints (ITT population)

From: Efficacy and Safety of Hizentra®, a New 20% Immunoglobulin Preparation for Subcutaneous Administration, in Pediatric Patients with Primary Immunodeficiency

Efficacy endpoint Children Adolescents Adults
2–11 years 12–15 years 16–64 years
N = 17 N = 5 N = 24
IgG trough levels
 Pre-study
 Mean ± SD 6.94 ± 1.223 7.99 ± 1.946 7.81 ± 1.666
 Median (range) 6.77 (5.3–10.1) 7.88 (5.4–10.3) 7.49 (5.3–11.7)
 Infusions 12–17a, e
 Mean ± SD 7.86 ± 1.720 7.91 ± 1.432 8.31 ± 1.250
 Median (range) 7.66 (5.1–12.4) 7.54 (6.2–9.5) 8.15 (6.3–10.9)
 Infusions 12–41f
 Mean ± SD 7.78 ± 1.510 8.14 ± 1.390 8.32 ± 1.211
 Median (range) 7.67 (5.2–11.2) 7.71 (6.7–9.9) 8.25 (6.4–10.8)
SBIs
 Number (%) of patientsb 0 (0) 0 (0) 0 (0)
 Number of events (annual rate per patient) 0 (0) 0 (0) 0 (0)
 [upper 99% CL] [0.511] [1.705] [0.376]
[0.543]d NA NA
All infections
 Number (%) of patientsb 15 (88.2) 3 (60.0) 18 (75.0)
 Number of events (annual rate per patient) 43 (4.77) 14 (5.18) 67 (5.47)
  40 (4.714)d NA NA
 [95% CI] [3.452; 6.426] [2.833; 8.695] [4.241; 6.949]
[3.368, 6.419]d NA NA
Days missed from school/day care
 Number (%) of patientsc 8 (47.1) 4 (80.0) 8 (33.3)
 Number of days (annual rate per patient) 139 (14.90) 5 (1.79) 54 (4.28)
  68 (7.74)d NA NA
Days hospitalized due to infection
 Number (%) of patientsc 3 (17.7) 0 (0) 1 (4.2)
 Number of days (annual rate per patient) 78 (8.36) 0 (0) 8 (0.63)
  15 (1.71)d NA NA
Days with antibiotics for infection prophylaxis or treatment
 Number (%) of patientsb 11 (64.7) 1 (20.0) 17 (70.8)
 Number of days (annual rate per patient) 442 (49.04) 1 (0.37) 727 (59.38)
  260 (30.64)d NA NA
  1. With the exception of IgG trough levels, data are for the efficacy period
  2. ITT intent-to-treat, N number of patients in age group, SBI serious bacterial infections, CL confidence limit, CI confidence interval, NA not applicable
  3. aPrimary efficacy endpoint
  4. bThe total number of days in the study was 3,290 (children), 986 (adolescents), and 4,469 (adults)
  5. cThe total number of days from patient diaries was 3,406 (children), 1,020 (adolescents), and 4,607 (adults)
  6. dData excluding the patient who suffered from recurrent pneumonias
  7. eData from a post hoc nonparametric analysis of the change in IgG levels from baseline to the efficacy period (Infusions 12–17): children (mean change from baseline, 0.920; Hodges–Lehmann point estimate, 0.680; two-sided 95% CI, 0.030, 1.500); adolescents (mean change from baseline, −0.089; Hodges–Lehmann point estimate, −0.152; two-sided 95% CI, −0.770, 0.810); adults (mean change from baseline, 0.366; Hodges–Lehmann point estimate, 0.377; two-sided 95% CI, −0.058, 0.838)
  8. fData from a post hoc nonparametric analysis of the change in IgG levels from baseline to the entire study period (Infusions 12–41): children (mean change from baseline, 0.846; Hodges–Lehmann point estimate, 0.683; two-sided 95% CI, 0.055, 1.445); adolescents (mean change from baseline, 0.145; Hodges–Lehmann point estimate, −0.020; two-sided 95% CI, −0.370, 1.300); adults (mean change from baseline, 0.386; Hodges–Lehmann point estimate, 0.355; two-sided 95% CI, −0.090, 0.820)