Table III.
Efficacy endpoints (ITT population)
| 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 | |
With the exception of IgG trough levels, data are for the efficacy period
ITT intent-to-treat, N number of patients in age group, SBI serious bacterial infections, CL confidence limit, CI confidence interval, NA not applicable
aPrimary efficacy endpoint
bThe total number of days in the study was 3,290 (children), 986 (adolescents), and 4,469 (adults)
cThe total number of days from patient diaries was 3,406 (children), 1,020 (adolescents), and 4,607 (adults)
dData excluding the patient who suffered from recurrent pneumonias
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)
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)