Table 4.
Clinical Characteristics and Post-Transplant Morbidities of Infants and Children Transplanted for Genetics Disorders of Surfactant Metabolism (1993–2015)
| Infants | Children | ||
|---|---|---|---|
|
| |||
| Number listed | 35 | 17 | |
|
| |||
| Number transplanted (% listed) | 28 (0.80) | 16 (0.94) | |
|
| |||
| Genetic etiology | |||
| SFTPB | 14 (0.40) | 0 (0) | <0.000 |
| SFTPC | 1 (0.03) | 11 (0.65) | 1 |
| ABCA3 | 17 (0.49) | 5 (0.29) | |
| NKX2-1 | 3 (0.08) | 1 (0.06) | |
|
| |||
| Sex Female | 19 (0.54) | 11 (0.65) | 0.48 |
| Male | 16 (0.46) | 6 (0.35) | |
|
| |||
| Race/Ethnicity | 0.57 | ||
| European descent | 27 (0.77) | 14 (0.82) | |
| African descent | 1 (0.03) | 2 (0.12) | |
| Hispanic | 2 (0.06) | 0 (0) | |
| Middle Eastern | 3 (0.08) | 0 (0) | |
| Other | 2 (0.06) | 1 (0.06) | |
|
| |||
| Death prior to transplantation | 7/35 (0.20) | 1/17 (0.06) | 0.25 |
|
| |||
| Wait time to transplant (months ± SD) | 1.9 ±1.5 | 9.3 ±11 | 0.014 |
|
| |||
| Mechanically ventilated at transplant | 25/28 (0.89) | 2/16 (0.13) | <0.0001 |
|
| |||
| ECMO prior to transplant (% listed) | 7/35 (0.20) | 1/17 (0.06) | 0.25 |
|
| |||
| Survival at 1 year post transplant | 23/28 (0.82) | 16/16 (1.0) | 0.14 |
|
| |||
| Survival at 5 years post transplant | 14/25 (0.56) * | 11/14 (0.79) * | 0.19 |
|
| |||
| CMV infection | 2/28 (0.07) | 2/16 (0.13) | 0.61 |
|
| |||
| Seizures | 3/28 (0.11) | 0/16 (0) | 0.29 |
|
| |||
| Hypertension by 5 years post transplant | 4/15 (0.27)† | 4/13 (0.31)† | 1.0 |
|
| |||
| Renal insufficiency by 5 years post transplant | 2/15 (0.13)‡ | 1/12 (0.08)‡ | 1.0 |
|
| |||
| Bronchiolitis obliterans by 5 years post transplant | 6/17 (0.35)§ | 8/14 (0.57)§ | 0.22 |
|
| |||
| PTLD by 5 years post transplant | 2/14 (0.14) | 1/11 (0.09) | 1.0 |
|
| |||
| Gastrostomy tube | 22/28 (0.79) | 14/16 (0.88) | 0.69 |
|
| |||
| Growth impairment | 14/26 (0.54)ǁ | 10/16 (0.63) | 0.58 |
|
| |||
| Speech delay | 19/22 (0.86)ǁ | 2/16 (0.13) | <0.0001 |
|
| |||
| Motor delay | 19/24 (0.79)ǁ | 2/16 (0.13) | <0.0001 |
|
| |||
| Hearing loss | 7/21 (0.33)ǁ | 0/16 (0) | 0.012 |
Data presented as number (percent) of individuals
*
Does not include infants or children alive, but not yet 5 years post-transplant
†
Numerator and denominator include patients with hypertension who died prior to 5 years post-transplant
‡
Numerator and denominator include patients with renal insufficiency who died prior to 5 years post-transplant
§
Numerator and denominator include patients with BO who died prior to 5 years post-transplant
ǁ
Information not available for all infants