Congenital cystic adenomatoid malformation of the lung (CCAM) and Bronchopulmonary sequestration (BPS): Prenatal diagnosis, pre- and postnatal interventions, and early- and long-term outcome (14 years clinical experience with 60 patients)

DEUTSCHE ZUSAMMENFASSUNG siehe PDF-Datei S. 7 (Teil I) und S. 111
(Teil II). /// ENGLISH ABSTRACT: BACKGROUND AND PURPOSE: Fetal
cystic lung lesions, such as congenital cystic adenomatoid
malformation of the lung (CCAM) and bronchopulmonary sequestration
(BPS), were once considered rare and of poor prognosis. The
advances in prenatal imaging and fetal therapeutic intervention
techniques have contributed to a revision of this opinion. However,
hardly any long-term investigation exists and prenatal predictive
factors are still controversially debated with a lack of detail.
This complicates adequate counseling of expectant parents and
patients management. The goal of this research was four-fold: (1)
to study the long-term outcome of children with prenatally detected
congenital cystic lung lesions, (2) to compare the long-term
outcome with the early outcome of the disease, (3) to determine
prenatal ultrasonographic features which are prognostic for the
outcomes, and (4) to compare the findings of this study with the
data obtained by an extended statistical analysis of all major
series of patients published in the literature. PATIENTS AND
METHODS: This prospective cohort study, a long-term follow-up
study, was conducted on 60 fetuses prenatally diagnosed with cystic
lung lesions and ultrasonographically evaluated from 1988 through
2002 at the University of California, San Francisco (UCSF) Medical
Center and at the University Hospital Großhadern of the
Ludwig-Maximilians-University (LMU) Munich, Germany. Based on a
follow-up questionnaire (see appendix of PDF-file for copy) with 39
questions, sent to the patients’ parents up to 14 years later, and
a review of the patients’ medical records, clinical data were
extracted about present and past respiratory and non-respiratory
symptoms, therapeutic interventions, and imaging studies.
Respiratory difficulties such as the requirement of ventilatory
support or oxygen, recurrent respiratory infections, asthma, and/or
limited physical endurance were categorized into four severity
groups. The study patients were classified into five surgical
intervention groups: (1) termination of pregnancy, (2) invasive
fetal treatment, (3) neonatal surgery, (4) childhood surgery, and
(5) no surgical intervention. Postoperative early and late
complications were investigated. The outcome was grouped into (i)
early neonatal outcome, (ii) interim childhood outcome, and (iii)
final outcome. The age at improvement was evaluated. To identify
prenatal predictors for the outcome, the children’s prenatal
ultrasound data were retrospectively reviewed. Data about the size,
(Stocker) type, and location of the lung lesion and the development
of a hydrops fetalis, mediastinal shift, ascites, pleural and
pericardial effusions, skin edema, placental thickening,
polyhydramnios, and the fetal growth were extracted. All
quantifiable parameters were categorized into four severity groups.
The size of the mass was categorized into (i) small, (ii) moderate,
(iii) large, or (iv) very large. The predictive value of each
prenatal parameter examined in this study was compared with
predictions published in the literature. All study data were
analyzed using the Pearson's chi-square test, t-tests of the means,
analysis of variance (ANOVA), and bivariate correlation. The
Committee of Human Research approved the study (see appendix of
PDF-file for approval letter). RESULTS: The survival rate in this
study of 60 fetuses with cystic lung lesions was 94% (51 of 54
cases) to the exclusion of 4 terminated pregnancies and 2 deceased,
untreated fetal treatment candidates. Three patients died after
birth despite invasive fetal treatment due to advanced hydrops
fetalis and delayed open fetal surgery. Of the 51 surviving
children, 11 (22%) children had invasive fetal treatment, 12 (24%)
were operated on as neonates, 15 (29%) were operated on in
childhood, and 13 (25%) had no surgery. Invasive fetal treatment
had a success rate of 79% (11/14) with hydrops regressions 1 to 3
weeks after intervention. Asymptomatic or only mildly affected
after birth were 31 (61%) children, 4 (8%) neonates had moderate
respiratory symptoms, and 16 (31%) had severe early respiratory
difficulties. Prematurity had a considerable influence on the early
respiratory difficulties (p