Congenital Diseases, Diaphragmatic Hernia, Pulmonary Hypoplasia
Conditions
Keywords
Isolated Congenital Diaphragmatic Hernia, Fetal Surgery, Pulmonary Hypoplasia
Brief summary
This trial investigates whether prenatal intervention improves survival rate of fetuses with isolated congenital diaphragmatic hernia and severe pulmonary hypoplasia, as compared to expectant management during pregnancy, both followed by standardized postnatal care.
Interventions
PROCEDUREfetal endoluminal tracheal occlusion
percutaneous fetoscopy, positioning of endoluminal balloon at 27-30 weeks and whenever possible elective removal at 34 weeks
OTHERwatchful waiting during pregnancy
pregnancy surveillance for fetal wellbeing, development of polyhydramnios and cervical shortening
Sponsors
King's College Hospital NHS Trust (UK)
Hospital Clinic of Barcelona
Hopital Antoine Beclere
University Hospital, Bonn
Mater Mothers' Hospital
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Ospedale Pediatrico Bambino Gesù, Rome (IT)
Mount Sinai Hospital, Canada
National Center for Child Health and Development, Tokyo (JP)
The University of Texas Health Science Center, Houston
Medical University of Warsaw
University Hospital, Gasthuisberg
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
FEMALE
Age
18 Years to 50 Years
Healthy volunteers
No
Inclusion criteria
* Patients aged 18 years or more, who are able to consent * Singleton pregnancy * Anatomically and chromosomally normal fetus * Left sided diaphragmatic hernia * Gestation at randomization prior to 29 wks plus 5 d (so that occlusion is done at the latest on 29 wks plus 6 d) * Estimated to have severe pulmonary hypoplasia, defined prenatally as: O/E LHR \<25 %, irrespective of the liver position * Acceptance of randomization and the consequences for the further management during pregnancy and thereafter. * The patients must undertake the responsibility for either remaining close to, or at the FETO center, or being able to travel swiftly and within acceptable time interval to the FETO center until the balloon is removed. * Intended postnatal treatment center must subscribe to suggested guidelines for standardized postnatal treatment. * Provide written consent to participate in this RCT
Exclusion criteria
* Maternal contraindication to fetoscopic surgery or severe medical condition in pregnancy that make fetal intervention risk full * Technical limitations precluding fetoscopic surgery, such as severe maternal obesity, uterine fibroids or potentially others, not anticipated at the time of writing this protocol. * Preterm labour, cervix shortened (\<15 mm at randomization) or uterine anomaly strongly predisposing to preterm labour, placenta previa * Patient age less than 18 years * Psychosocial ineligibility, precluding consent * Diaphragmatic hernia: right-sided or bilateral, major anomalies, isolated left-sided outside the O/E LHR limits for the inclusion criteria * Patient refusing randomization or to comply with return to FETO center during the time period the airways are occluded or for elective removal of the balloon
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Survival at discharge from neonatal intensive care unit | at discharge from neonatal intensive care unit | The baby can be discharged either alive (and then it qualifies as surviving at discharge) or dead. Not discharged babies have not reached the primary endpoint. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| grading of oxygen dependency | born >32 wks: between 28-56d of life; born <32wks: 36wks postmenstrual age | — |
| occurrence of pulmonary hypertension | within first weeks of life | determined by cardiac ultrasound |
| number of days in Neonatal Intensive Care Unit (NICU) | within hospital stay | As long as the baby is in the hospital, it is hospitalized either in NICU or in another, less intensive care unit. The number of days in NICU is an outcome variable, expressed in days. |
| number of days of ventilatory support | within NICU stay | — |
| presence of periventricular leucomalacia | 2 months of life | — |
| presence of neonatal sepsis, intraventricular haemorrhage, retinopathy grade III or higher | within hospital stay | — |
| number of days till full enteral feeding | within first 2 years of life | — |
| prenatal increase in lung volume after FETO | prior to balloon removal | volume of lung after occlusion |
| day of surgery | within hospital stay | — |
| requirement for use of patch for repair | at the time of postnatal surgery | — |
| bronchopulmonary dysplasia | with the first 8 weeks | defined as oxygen need for at least 28 days |
| Need for Extracorporeal membrane oxygenation | during NICU admission | — |
| Defect size | at the time of postnatal surgery | — |
| number of days alive in case of postnatal death | during NICU admission | — |
| presence of gastro-esophagal reflux | at discharge | — |
Countries
Australia, Belgium, Canada, France, Germany, Italy, Japan, Poland, Spain, United Kingdom, United States
Outcome results
None listed