TABLE 2.
Variable | Overall | Randomized to ETV+CPC | Randomized to VPS | p Value | Difference (95% CI) for ETV+CPC vs VPS |
---|---|---|---|---|---|
Head circumference in cm | 49.5 (46.9, 51.5) | 50.0 (48.2, 52.9) | 48.0 (46.1, 51.3) | 0.008 | 2.0 (0.5, 3.4) |
Treatment failure | 33/100 (33.0%) | 18/51 (35.3%) | 15/49 (30.6%) | 0.67 | 4.7% (−13.7%, 23.1%) |
Infection | 4 (4.0%) | 2 (3.9%) | 2 (4.1%) | >0.99 | −0.2% (−7.8%, 7.5%) |
All-cause mortality | 15 (15.0%) | 9 (17.6%) | 6 (12.2%) | 0.58 | 5.4% (−8.5%, 19.3%) |
BSID-III scaled scores | |||||
Cognitive, primary outcome | 4 (1, 5) | 4 (1, 6) | 3 (1, 5) | 0.17 | 0 (0, 2) |
Gross motor | 1 (1, 5) | 1 (1, 9) | 1 (1, 3) | 0.07 | 0 (0, 1) |
Fine motor | 5 (1, 8) | 6 (1, 9) | 5 (1, 7) | 0.31 | 0 (0, 2) |
Change in BSID-III scaled scores from baseline | |||||
Cognitive | 0 (−2, 3) | 1 (−2, 4) | 0 (−2, 3) | 0.64 | 0 (−1, 2) |
Gross motor | 0 (−3, 1) | 0 (−3, 4) | 0 (−2, 0) | 0.62 | 0 (−1, 2) |
Fine motor | 2 (−2, 4) | 2 (−3, 4) | 1 (−2, 4) | 0.67 | 0 (−2, 1) |
Brain vol: raw, in cm3 | |||||
At baseline | 448 (379, 514) | 435 (379, 510) | 461 (382, 518) | 0.84 | −5 (−43, 34) |
At 12 mos | 813 (709, 941) | 809 (692, 937) | 821 (741, 956) | 0.21 | −33 (−97, 19) |
At 24 mos | 831 (725, 923) | 774 (688, 920) | 863 (740, 948) | 0.36 | −30 (−95, 30) |
Brain vol: normalized for age and sex | |||||
Normalized brain vol at 24 mos, z-score | −2.6 (−3.9, −1.4) | −3.0 (−4.1, −1.4) | −2.4 (−3.6, −1.4) | 0.40 | −0.3 (−1.1, 0.5) |
Normal brain vol at 24 mos | 16/90 (17.8%) | 9/45 (20.0%) | 7/45 (15.6%) | 0.78 | 4.0% (−11.0%, 20.0%) |
Substantial brain growth from baseline to 24 mos | 23/89 (25.8%) | 7/44 (15.9%) | 16/45 (35.6%) | 0.05 | −20.0% (−38.0%, −1.0%) |
Substantial brain vol loss from baseline to 24 mos | 19/89 (21.3%) | 9/44 (20.5%) | 10/45 (22.2%) | >0.99 | −2.0% (−19.0, 15.0%) |
Substantial brain vol loss from 12 to 24 mos | 69/90 (76.7%) | 34/45 (75.6%) | 35/45 (77.8%) | >0.99 | −2.0% (−20.0%, 15.0%) |
Substantial brain growth from 12 to 24 mos | 2/90 (2.2%) | 1/45 (2.2%) | 1/45 (2.2%) | >0.99 | −0% (−6.0%, 6.0%) |
Data are expressed as median (interquartile range) or number (percent), unless indicated otherwise. The p values were derived from the Mann-Whitney U-test or Fisher’s exact test. Twenty-four–month data for head circumference were available for 90 patients (45 ETV+CPC, 45 VPS), and data for BSID-III scores and volumes were available for 89 patients (44 ETV+CPC, 45 VPS). Hodges-Lehmann estimates and 95% confidence intervals were used for differences in medians and normal approximation for differences in proportions.