Table 2.
Infection Status and Age at Transplant, by Trigger for Diagnosis
| Time interval | Variable | Trigger for diagnosisa | p value | ||
|---|---|---|---|---|---|
| Family history | Newborn screening | Clinical illnessb | |||
| 1982–2018 | Total patients (n) | 257 | 130 | 505 | |
| Infection status at HCT, n (%) | <0·001 | ||||
| Infected/Active | 62 (24·1) | 27 (20·8) | 284 (56·2) | ||
| Infected/Resolved | 57 (22·2) | 17 (13·1) | 146 (28·9) | ||
| Infected/Unknownc | 5 (1·9) | 0 (0·0) | 18 (3·6) | ||
| No infection | 133 (51·8) | 86 (66·2) | 56 (11·1) | ||
| Unknown | 0 (0·0) | 0 (0·0) | 1 (0·2) | ||
| Age at HCT – actual | |||||
| Days of life, median (IQR) | 67·0 (29·0–151·0) | 92·5 (67·0–114·0) | 222·0 (167·0–311·0) | <0·001 | |
| Age at HCT – categorical n (%) | |||||
| <3·5 months old | 161 (62·6) | 87 (66·9) | 42 (8·3) | <0·001 | |
| ≥3·5 months old | 96 (37·4) | 43 (33·1) | 463 (91·7) | ||
| 2010–2018 | Total patients (n) | 49 | 130 | 89 | |
| Infection status at HCT, n (%) | |||||
| Infected/Active | 6 (12·2) | 27 (20·8) | 49 (55·1) | <0·001 | |
| Infected/Resolved | 11 (22·5) | 17 (13·1) | 24 (27·0) | ||
| Infected/Unknownc | 0 (0·0) | 0 (0·0) | 1 (1·1) | ||
| No Infection | 32 (65·3) | 86 (66·1) | 14 (15·7) | ||
| Unknown | 0 (0·0) | 0 (0·0) | 1 (1·1) | ||
| Age at HCT – actual | |||||
| Days of life, median (IQR) | 76·0 (49·0–132·0) | 92·5 (67·0–114·0) | 252·0 (152·0–461·0) | <0·001 | |
| Age at HCT – categorical, n (%) | |||||
| <3·5 months old | 31 (63·3) | 87 (66·9) | 8 (9·0) | <0·001 | |
| ≥3·5 months old | 18 (36·7) | 43 (33·1) | 81 (91·0) | ||
Infection status at HCT was evaluated based on trigger for diagnosis, with a sub-analysis (2010–2018) for the era when NBS became available.
a
Ten patients had an unknown single trigger for diagnosis identified and are therefore excluded from this analysis.
b
Typically related to infections, autoimmunity, and/or failure to thrive.
c
An infection pre-HCT was documented, but whether it was still active or had resolved at time of HCT was not recorded.
Abbreviations: FH, family history; IQR, interquartile range; NBS, newborn screening