Morbidity and Outcomes
Table 3 summarizes the clinical status of enrolled subjects at
time of Registry enrollment using Fan severity of illness
score18. While a quarter of subjects were
asymptomatic, the lifetime morbidity experienced by the children in this
cohort is substantial. Overall, 63% of subjects had a history of home
supplemental oxygen use, and 12% required chronic (>3
weeks) invasive mechanical ventilation, with an additional 13%
requiring chronic non-invasive ventilatory support. Failure to thrive
occurred in 46% of subjects during their clinical course.
For the 527 subjects with outcome data available, 31 (6%) have died
since enrollment. The diagnoses of those who died were immune-mediated
or connective tissue disease (n=10), bronchiolitis obliterans (n=6),
unclassified (n=4), other specific or multisystem disorders (n=4),
surfactant dysfunction (n=2), environmental/toxic/drug related (n=2),
lung developmental dysplasia (n=1), alveolar growth disorder (n=1), and
pulmonary alveolar proteinosis (n=1). No deaths occurred in children
with NEHI. Sixteen subjects in the registry underwent lung transplant
with primary indicated diagnoses of surfactant metabolic dysfunction
(n=5), bronchiolitis obliterans (n=3), unclassified chILD (n=3),
alveolar growth disorder (n=2), lung developmental dysplasia (n=1),
chILD associated with connective tissue or immune-mediated disorders
(n=1), and other specific or multisystem disorder (n=1). Four deaths
occurred among lung transplant recipients.