Congenital Diaphragmatic Hernia
Conditions
Brief summary
The purpose of this research is to determine if babies who undergo a Fetoscopic Endoluminal Tracheal Occlusion (FETO) procedure survive more often and have less long-term complications than babies who have similarly severe Congenital Diaphragmatic Hernia (CDH) that have not had the FETO procedure performed during pregnancy
Interventions
Mothers who chose to undergo a Fetoscopic Endoluminal Tracheal Occlusion procedure for severe left and right congenital diaphragmatic hernia will be followed until the infant is 2 years of age.
OTHERStandard of Care
Mothers who chose to receive expectant management for severe left and right congenital diaphragmatic hernia will be followed until the infant is 2 years of age.
Sponsors
Mayo Clinic
Study design
Observational model
CASE_CONTROL
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
FEMALE
Age
18 Years to 50 Years
Healthy volunteers
No
Inclusion criteria
* Singleton pregnancy * Normal fetal karyotype with confirmation by culture results, CMA with non-pathological variants, WES or WGS. Results by fluorescence in situ hybridization (FISH) will be acceptable if the patient is \> 26 weeks * Gestational age at enrollment prior to 29 6/7 weeks * Intrathoracic liver herniation Isolated left CDH with O/E LHR \< 30% at enrollment (18 0/7 to 29 5/7 weeks.) Isolated right CDH with O/E LHR \< 45% at enrollment (18 0/7 to 29 5/7 weeks). * Cervical length by transvaginal ultrasound \> 20 mm within 24 to 48 hours prior to FETO procedure * Patient meets psychosocial criteria * Informed consent understood
Exclusion criteria
* Multi-fetal pregnancy * History of natural rubber latex allergy * Preterm labor, cervix shortened (\<20 mm at enrollment or within 24 to 48 hours of FETO balloon insertion procedure) or uterine anomaly strongly predisposing to preterm labor, placenta previa * Psychosocial ineligibility, precluding consent: * Inability to reside within 30 minutes of Mayo Clinic. and inability to comply with the travel for the follow-up requirements of the trial * Patient does not have a support person (e.g., spouse, partner, mother) available to stay with the patient for the duration of the pregnancy at Mayo Clinic. * Bilateral CDH, isolated left sided CDH with O/E LHR 30% (measured at 180 to 295 weeks), isolated right sided CDH with O/E LHR \> 45% (measured at 180 to 295 weeks), as determined by ultrasound. * No intrathoracic liver herniation * Additional fetal anomaly and chromosomal abnormalities, associated anomalies recognized to alter survival prognosis (i.e., congenital heart disease) or presence of an underlying genetic syndrome (i.e., Fryns) by ultrasound, MRI, or echocardiogram at the fetal treatment center. * Maternal contraindication to fetoscopic surgery or severe maternal medical condition in pregnancy * History of incompetent cervix with or without cerclage * Placental abnormalities (previa, abruption, accreta) known at time of enrollment * Maternal-fetal Rh isoimmunization, Kell sensitization or neonatal alloimmune thrombocytopenia affecting the current pregnancy * Maternal HIV, Hepatitis-B, Hepatitis-C status positive because of the increased risk of transmission to the fetus during maternal-fetal surgery. If the patient's HIV or Hepatitis status is unknown, the patient must be tested and found to have negative results before enrollment * Uterine anomaly such as large or multiple fibroids or Mullerian duct abnormality * There is no safe or technically feasible fetoscopic approach to balloon placement * Participation in another intervention study that influences maternal and fetal morbidity and mortality or participation in this trial in a previous pregnancy
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of FETO procedure complications | 2 years | FETO procedure complications are defined as device dislodgement, intraoperative injury to fetus, procedural hemorrhage, post-procedural hemorrhage, preterm delivery, chorioamnion separation, chorioamnionitis, polyhydramnios, oligohydramnios, failed percutaneous or fetoscopic removal requiring cesarean section or EXIT procedure for removal, neonatal death due to asphyxia (if delivery before FETO removal), non-reassuring fetal heart rate monitoring. |
| Neonatal survival to discharge | 2 years | Number of neonates to survive from delivery to hospital discharge |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Presence of pulmonary hypertension | 6, 12, 18, and 24 months of age | Number infants to be diagnosed with pulmonary hypertension. Pulmonary hypertension is a determination of the ratio of the tricuspid regurgitation jet (estimation of the right ventricular systolic pressure) to systemic blood. Will be record as absent/none, systemic or suprasystemic |
| Need for supplemental oxygen | 6, 12, 18, and 24 months of age | Number of infants to require supplemental oxygen |
| Periventricular leukomalacia at < 2 months postnatally | 2 months | Number of infants to be diagnosed with periventricular leukomalacia at \< 2 months postnatally |
| Intraventricular hemorrhage (grade 0-III) | 6, 12, 18, and 24 months of age | Number of infants to be diagnosed with intraventricular hemorrhage (grade 0-III) |
| Retinopathy of prematurity (grade 3 or higher) | 6, 12, 18, and 24 months of age | Number infants to be diagnosed with retinopathy of prematurity (grade 3 or higher) |
| Gastro-esophageal reflux | 6, 12, 18, and 24 months of age | Number of infants to be diagnosed with gastro-esophageal reflux |
| Number of infant hospital readmissions | 6, 12, 18, and 24 months of age | Number of infants readmitted to hospital after initial discharge |
| Childhood growth failure | 6, 12, 18, and 24 months of age | Number of infants to experience childhood growth failure: weight gain velocity or decline \>2 in weight, for length Z score. |
| Recurrence of Congenital Diaphragmatic Hernia (CDH) repair | 6, 12, 18, and 24 months of age | Number of infants to require additional congenital diaphragmatic hernia (CDH) repair procedures. |
| Bowel obstruction | 6, 12, 18, and 24 months of age | Number of infants to experience a bowel obstruction. |
| Neurodevelopmental delay | 6, 12, 18, and 24 months of age | Number of infants to experience neurodevelopmental delay: Bayley Scales of Infant and Toddler Development-III(BSID)\>2 SD below mean, or other categorization at 24 months. |
| Bronchopulmonary dysplasia | 6, 12, 18, and 24 months of age | Number of infants to be diagnosed with bronchopulmonary dysplasia (BPD). It is defined according to the mode of respiratory support administered at 36 weeks' postmenstrual age, regardless of the prior duration or current level of oxygen therapy. The optimal diagnostic criteria will be no BPD, no support; grade 1 BPD, nasal cannula S2 L/min; grade 2 BPD, nasal cannula \>2 L/min or noninvasive positive airway pressure; and grade 3 BPD, invasive mechanical ventilation. |
Countries
United States
Contacts
PRINCIPAL_INVESTIGATORMauro Schenone, MD
Mayo Clinic
Outcome results
None listed