This trial will test whether temporary fetoscopic endoluminal tracheal occlusion (FETO) rather than expectant management during pregnancy, followed by standardized postnatal management, increases survival at discharge and decreases oxygen need at 6 months in case of survival till discharge.
This is a multi-center, non-blinded randomized controlled trial in fetuses with isolated moderate CDH, i.e. moderate lung hypoplasia (as determined by prenatal assessment of lung development). It essentially compares fetal therapy added to conventional postnatal care, versus expectant prenatal management during pregnancy followed by conventional postnatal care. Enrollment: Following completion of an inclusion/exclusion criteria checklist and obtaining informed consent, the subject will be randomized into two groups ("FETO" and "expectant"). Procedures: Group I: Standardized postnatal care (expectant group): mothers will be expectantly managed during pregnancies and babies receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. Group II: Prenatal intervention (FETO group): patients will undergo fetoscopic tracheal occlusion and ideally prenatal reversal of the occlusion followed by standardized postnatal care as in I. In this study FETO is to be done between 30 weeks plus 0 day and 31 weeks plus 6 days and removal of the balloon at 34 weeks plus 0 day to 34 weeks plus 6 days. This study trial is a pragmatical or efficacy trial: ideally mothers will deliver after removal of the balloon at those tertiary centers, typically offering postnatal care for the patient involved. In group II (FETO-group), mothers will, in between placement and removal of the balloon, thus carrying a fetus with obstructed airways, ideally remain under the care of our local fetal treatment center (further referred to as FETO center). As many as possible precautions are taken to avoid problems with balloon removal in case of earlier than expected delivery. * Balloons are to be electively removed prior to 35 weeks. FETO centers will provide 24/24 hours and 7/7 days services for management of fetuses with obstructed airways, either in utero or during labor and delivery. * Patients in the study and randomized to FETO, will be encouraged to stay near the FETO center. After reversal of the occlusion the patient will be referred to the tertiary care center where delivery and postnatal care will be undertaken. If the patient is not remaining at or close to the FETO center, the postnatal treatment center should organize likewise EXIT services.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
5
Placement of the balloon using the plug/unplug method.
The catheter assists with implanting the balloon in the plug/unplug method.
After birth, the babies will receive standardized postnatal care at a tertiary center used to manage babies with CDH. The recommendation is that they adhere to consensus guidelines published on the study website. For detailed description on this please visit https://www.karger.com/Article/Abstract/320622
Baylor College of Medicine/Texas Children's Hospital
Houston, Texas, United States
Proportion of Neonate Survival at Discharge From Hospital
The null hypothesis to be tested is that there is no difference in survival between fetuses managed expectantly during pregnancy versus those undergoing antenatal therapy (FETO).
Time frame: At hospital discharge, an average of 1.5 months
Participants Requiring Supplemental Oxygen
The number of survivors requiring supplemental oxygen at 6 months of age
Time frame: At 6 months of age
Grade of Oxygen Dependency
Measured as FiO2 (oxygen) amount required as a grade (0-III) with Grade 0 indicating the best outcome and Grade III indicating the worst outcome. Grade 0 = No Bronchopulmonary Dysplasia (BPD); Grade I = FiO2 21% or room air; Grade II = FiO2 22-29%; Grade III = FiO2 \>29%, CPAP or mechanical ventilation.
Time frame: at 6 months of age
Occurrence of Severe Pulmonary Hypertension
Occurrence of severe pulmonary hypertension in the neonatal period.
Time frame: During the first 4 weeks of life (neonatal period).
ECMO (Extracorporeal Membrane Oxygenation) Support
Need for extracorporeal membrane oxygenation (ECMO) in the neonatal period
Time frame: Neonatal period (during the first 4 weeks of life)
CDH Defect Size
Postnatal grade classification (A-D) using CDH study group standardized system with A being the smallest defects and D being the largest defects. A = Defect entirely surrounded by muscle; B = Small (\<50%) portion of the chest wall devoid of diaphragm tissue; C = Large (\>50%) portion of the chest wall devoid of diaphragm tissue; D = Complete or near complete absence of the diaphragm.
Time frame: Measured in neonate at delivery by MRI and/or ultrasound
Number of Days in the NICU
Length of stay in the neonatal intensive care unit measured in days
Time frame: At the time of discharge from the NICU, an average of 1.5 months
Ventilatory Support
Length of time participants required ventilator support measured in days.
Time frame: During the first 4 weeks of life (neonatal period)
Number of Subjects With Periventricular Leukomalacia (PVL)
As measured by presence in medical record ≤ 2 months postnatally by ultrasound (yes/no)
Time frame: During first 2 months of life
Neonatal Sepsis
As measured by presence in medical record
Time frame: During the first 4 weeks of life (neonatal period)
Intraventricular Hemorrhage
Measured as presence in neonate during first month by MRI and/or ultrasound.
Time frame: During first month of life
Retinopathy of Prematurity
Postnatal grade classification presence of grade III or higher using standardized system (yes/no)
Time frame: At the time of discharge from the NICU, an average of 1.5 months
Days to Full Enteral Feeding
The number of days until full enteral feeding
Time frame: At hospital discharge, an average of 1.5 months
Gastroesophageal Reflux
Presence of reflux above 1/3 of esophagus on clinically indicated radiologic exam
Time frame: At the time of discharge from the NICU, an average of 1.5 months
CDH (Congenital Diaphragmatic Hernia) Surgery Repair
How many days from birth until the surgery is performed to repair the defect.
Time frame: From the time of birth until discharge from the NICU
Use of Patch in CDH Repair
The number of participants who had a patch used in the repair of the CDH defect.
Time frame: At the time of the surgical repair postnatally, up to 3 days postnatal
Survival
The number of participants that survived to 24 months of age
Time frame: At 24 months of age
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