Table 3.
Months (mean ± SD) | |||
---|---|---|---|
diagnosis | treatment | death | |
SCID infants identified early+ (n=7) |
1.0 ± 0 | 3.7 ± 4.3 | all alive |
| |||
SCID infants identified late− (n=32) |
9.0 ± 7.6 | 9.6 ± 5.4 | 17.6 ± 10.4% |
SCID infants with HCT (n=23) |
6.9 ± 5.0 | 9.8 ± 5.5 | 17.3 ± 7.5** |
SCID infants with no HCT (n=8) |
15.4 ± 10.3 | 19.4 ± 14.0 | |
SCID infant with PEG- ADA therapy^ (n=1) |
7 | 7 | 8 |
Based on survey responses from physicians and families of SCID children.
Identified early is based on known family history of SCID, prior to manifestation of infections.
Identified late is defined as confirmed with SCID after manifestation of infections.
Twenty out of 32 SCID patients identified late died.
Ten out of the 23 SCID patients transplanted died.
All eight SCID patients without HCT died.
PEG-ADA, polyethylene glycol-modified adenosine deaminase enzyme replacement, specifically for SCID with adenosine deaminase deficiency