Fetal Tracheal Balloon Study in Diaphragmatic Hernia
| Status: | Completed |
|---|---|
| Conditions: | Gastrointestinal, Pulmonary |
| Therapuetic Areas: | Gastroenterology, Pulmonary / Respiratory Diseases |
| Healthy: | No |
| Age Range: | 18 - Any |
| Updated: | 4/13/2015 |
| Start Date: | September 2008 |
| End Date: | January 2015 |
| Contact: | Francois I Luks, MD, PhD |
| Email: | Francois_Luks@brown.edu |
| Phone: | 401-228-0556 |
Phase 2 Fetal Tracheal Balloon (IDE G080077) Study in Diaphragmatic Hernia
The purpose of this phase 2 limited study is to examine whether prenatal intervention
correct the lung underdevelopment associated with severe diaphragmatic hernia.
correct the lung underdevelopment associated with severe diaphragmatic hernia.
Congenital diaphragmatic hernia (CDH) has traditionally been associated with very high
mortality rates. Most infants died of pulmonary hypoplasia and severe pulmonary
hypertension. This led to correction of CDH and pulmonary hypoplasia before birth.
Unfortunately, maternal morbidity of open fetal surgery was significant and fetal mortality
was very high (>60%). Moreover, the results of postnatal therapy for CDH improved
dramatically, from less than 20% survival several decades ago to more than 70% today.
Fetal intervention has evolved as well, to a minimally invasive approach that involves a
single endoscopic port and occlusion of the fetal trachea. While this has considerably
decreased the morbidity and fetal mortality of the in utero procedure, its results do not
exceed the overall (i.e., non-stratified) results of contemporary postnatal treatment. Most
recently, a multicentric cooperative study under (Eurofoetus) has conducted a clinical trial
comparing postnatal treatment with endoscopic fetal tracheal occlusion for the most severe
forms of CDH. Results of the Eurofoetus trial and of a recent retrospective review involving
European and North-American centers have shown the following: 1) It is possible to identify
a specific subgroup of fetuses with CDH in whom survival can be predicted to be less than
10%, despite all current methods of postnatal treatment, 2) Survival of fetuses with
predicted postnatal survival of 8% was >50% following endoscopic fetal tracheal occlusion,
and 3) Fetal tracheal occlusion in that group resulted in an increase in lung size (LHR),
from an average of 0.7 pre-intervention, to 1.7 post-intervention.
Based on the available research literature, the results of the Eurofoetus trial, and this
institution's experience with endoscopic fetal surgery, we hypothesize that in the highest
risk group of fetuses with congenital diaphragmatic hernia, where chances of survival is
estimated at less than 10%, endoscopic fetal tracheal occlusion in late second trimester,
with reversal of occlusion in mid-third trimester, allows catch-up lung growth and
maturation and converts the condition into one with intermediate to good prognosis
(predicted survival 50-60%). We propose to offer this form of treatment, under an
FDA-approved Investigational Device Exemption (G080077), to eligible patients, on a
case-by-case basis, after discussion before a multidisciplinary board.
mortality rates. Most infants died of pulmonary hypoplasia and severe pulmonary
hypertension. This led to correction of CDH and pulmonary hypoplasia before birth.
Unfortunately, maternal morbidity of open fetal surgery was significant and fetal mortality
was very high (>60%). Moreover, the results of postnatal therapy for CDH improved
dramatically, from less than 20% survival several decades ago to more than 70% today.
Fetal intervention has evolved as well, to a minimally invasive approach that involves a
single endoscopic port and occlusion of the fetal trachea. While this has considerably
decreased the morbidity and fetal mortality of the in utero procedure, its results do not
exceed the overall (i.e., non-stratified) results of contemporary postnatal treatment. Most
recently, a multicentric cooperative study under (Eurofoetus) has conducted a clinical trial
comparing postnatal treatment with endoscopic fetal tracheal occlusion for the most severe
forms of CDH. Results of the Eurofoetus trial and of a recent retrospective review involving
European and North-American centers have shown the following: 1) It is possible to identify
a specific subgroup of fetuses with CDH in whom survival can be predicted to be less than
10%, despite all current methods of postnatal treatment, 2) Survival of fetuses with
predicted postnatal survival of 8% was >50% following endoscopic fetal tracheal occlusion,
and 3) Fetal tracheal occlusion in that group resulted in an increase in lung size (LHR),
from an average of 0.7 pre-intervention, to 1.7 post-intervention.
Based on the available research literature, the results of the Eurofoetus trial, and this
institution's experience with endoscopic fetal surgery, we hypothesize that in the highest
risk group of fetuses with congenital diaphragmatic hernia, where chances of survival is
estimated at less than 10%, endoscopic fetal tracheal occlusion in late second trimester,
with reversal of occlusion in mid-third trimester, allows catch-up lung growth and
maturation and converts the condition into one with intermediate to good prognosis
(predicted survival 50-60%). We propose to offer this form of treatment, under an
FDA-approved Investigational Device Exemption (G080077), to eligible patients, on a
case-by-case basis, after discussion before a multidisciplinary board.
Inclusion Criteria:
- Singleton pregnancies
- Isolated congenital diaphragmatic hernia
- Normal karyotype (amniocentesis)
- Initial diagnosis before 26 weeks gestation
- Competent cervix
- Severity of CDH: lung-to-head ratio (LHR) ≤0.8 at 22-26 weeks gestation
- Liver herniation in the chest
- Informed consent
Exclusion Criteria:
- Preterm labor, premature rupture of membranes or amniotic leak
- Significant maternal morbidity
- Minor (<18 years)
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