Fetal myelomeningocele (fMMC) repair has become accepted as a standard of care option in selected circumstances. We reviewed our outcomes for fMMC repair from referral and evaluation through surgery, ...delivery and neonatal discharge.
All patients referred for potential fMMC repair were reviewed from January 1, 2011 through March 7, 2014. Maternal and neonatal data were collected on the 100 patients who underwent surgery.
29% of those evaluated met the criteria and underwent fMMC repair (100 cases). The average gestational age was 21.9 weeks at evaluation and 23.4 weeks at fMMC repair. Complications included membrane separation (22.9%), preterm premature rupture of membranes (32.3%) and preterm labor (37.5%). Average gestational age at delivery was 34.3 weeks and 54.2% delivered at ≥35 weeks. The perinatal loss rate was 6.1% (2 intrauterine fetal demises and 4 neonatal demises); 90.8% of women delivered at the Children's Hospital of Philadelphia and 3.4% received transfusions. With regard to the neonates, 2 received ventriculoperitoneal shunts prior to discharge; 71.1% of neonates had no evidence of hindbrain herniation on MRI. Of the 80 neonates evaluated, 55% were assigned a functional level of one or more better than the prenatal anatomic level.
In an experienced program, maternal and neonatal outcomes for patients undergoing fMMC repair are comparable to results of the MOMS trial.
To describe our experience with treprostinil, evaluate correlations with cardiac function, and assess for adverse effects in neonates with congenital diaphragmatic hernia-related pulmonary ...hypertension (CDH-PH).
A retrospective review of a single-center prospective registry at a quaternary care children's hospital. Patients included in the study had CDH-PH treated with treprostinil between April 2013 and September 2021. Assessed outcomes were brain-type natriuretic peptide levels and quantitative echocardiographic parameters collected at baseline, 1 week, 2 weeks, and 1 month after treprostinil initiation. Right ventricular (RV) function was assessed by tricuspid annular plane systolic excursion Z-score and speckle tracking echocardiography (global longitudinal and free wall strain). Septal position and left ventricular (LV) compression were assessed by eccentricity index and M-mode Z-scores.
Fifty-one patients were included, with an average expected/observed lung-to-head ratio of 28.4 ± 9.0%. Most patients required extra-corporeal membrane oxygenation (n = 45, 88%). Survival to hospital discharge was 31/49 (63%). Treprostinil was initiated at a median age of 19 days with a median effective dose of 34 ng/kg/minute. Median baseline brain-type natriuretic peptide level decreased from 416.9 pg/mL to 120.5 pg/mL after 1 month. Treprostinil was associated with improved tricuspid annular plane systolic excursion Z-score, RV global longitudinal strain, RV free wall strain, LV eccentricity index, and LV diastolic and systolic dimensions, reflecting less compression by the RV, regardless of ultimate patient survival. No serious adverse effects were recorded.
In neonates with CDH-PH, treprostinil administration is well tolerated and is associated with improved RV size and function.
Prenatal management of congenital diaphragmatic hernia Danzer, Enrico; Rintoul, Natalie E.; van Meurs, Krisa P. ...
Seminars in fetal & neonatal medicine,
December 2022, 2022-12-00, 20221201, Letnik:
27, Številka:
6
Journal Article
Recenzirano
Recently, two randomized controlled, prospective trials, the Tracheal Occlusion to Accelerate Lung Growth (TOTAL) trials, reported the outcomes on fetal endoluminal tracheal occlusion (FETO) for ...isolated left congenital diaphragmatic hernia (CDH). FETO significantly improved outcomes for severe hypoplasia. The effect in moderate cases, where the balloon was inserted later in pregnancy, did not reach significance. In a pooled analysis investigating the effect of the heterogeneity of the treatment effect by the time point of occlusion and severity, the difference may be explained by a difference in the duration of occlusion. Nevertheless, FETO carries a significant risk of preterm birth. The primary objective of this review is to provide an overview of the rationale for fetal intervention in CDH and the results of the randomized trials. The secondary objective is to discuss the technical aspects of FETO. Finally, recent developments of potential alternative fetal approaches will be highlighted.
•Congenital diaphragmatic hernia (CDH) is a prenatally diagnosed malformation that is associated with significant neonatal mortality and long-term morbidities.•Following prenatal diagnosis, families should receive individualized counseling regarding mid gestational management options based on prenatal and associated prognostic measures.•The randomized trials of Tracheal Occlusion to Accelerate Lung Growth (TOTAL) provided level one evidence that fetoscopic endoluminal tracheal occlusion (FETO) improves survival in selected cases of isolated CDH.•Fetal surgical intervention is however associated with maternal and fetal risks.•Continued evaluation of children enrolled in the TOTAL trials will be necessary to evaluate the long-term impact of fetal intervention and contribution of prematurity to morbidity outcomes.•Prior to widespread implementation of FETO to less experienced centers, prospective registration of new cases treated with FETO at high volume centers with experience in both fetal therapy and CDH care are needed to further increase our understanding of the effects FETO on the natural history of CDH.•Development of a pulmonary hypoplasia “biophysical profile” that incorporates measures of both lung size and pulmonary vascular function is needed to improve risk stratification for prenatal counseling and selection for fetal intervention.•Technical refinements and investigation of additional, but preferentially alternative, non-surgical prenatal management options, such as transplacental pharmacologic modulation of lung development or cell-based and tissue engineering therapy may improve outcomes and decrease the risk to the mother and fetus.
To compare outcomes between low birth weight (LBW; <2.5 kg) and standard birth weight neonates undergoing cardiac surgery.
A single-center retrospective study of neonates undergoing cardiac surgery ...with cardiopulmonary bypass from 2012 to 2018. LBW neonates were 1:2 propensity score–matched to standard birth weight neonates (n = 93 to n = 186) using clinical characteristics. The primary and secondary outcomes were survival to hospital discharge and postoperative complications, respectively. After matching, regression analyses were conducted to compare outcomes.
The LBW group had a higher proportion of premature neonates than the standard birth weight group (60% vs 8%; P < .01) and were less likely to survive to hospital discharge (88% vs 95%; OR, 0.39; 95% CI, 0.15-0.97). There was no difference in unplanned cardiac reoperations or catheter-based interventions, cardiac arrest, extracorporeal membrane oxygenation, infection, and end-organ complications between the groups. Among LBW infants, survival was improved at weight >2 kg.
LBW is a risk factor for decreased survival. LBW neonates weighing >2 kg have survival comparable to those weighing >2.5 kg.
Extracorporeal membrane oxygenation (ECMO) is a universally accepted and life-saving therapy for neonates with respiratory or cardiac failure that is refractory to maximal medical management. Early ...studies found unacceptable risks of mortality and morbidities such as intracranial hemorrhage among premature and low birthweight neonates, leading to widely accepted ECMO inclusion criteria of gestational age ≥34 weeks and birthweight >2 kg. Although contemporary data is lacking, the most recent literature demonstrates increased survival and decreased rates of intracranial hemorrhage in premature neonates who are supported with ECMO. As such, it seems like the right time to push the boundaries of ECMO on a case-by-case basis beginning with neonates 32–34 weeks GA in large volume centers with careful neurodevelopmental follow-up to better inform practices changes on this select population.
To evaluate associations between cardiac catheterization (cath) hemodynamics, quantitative measures of right ventricular (RV) function by echocardiogram, and survival in patients with congenital ...diaphragmatic hernia (CDH).
This single-center retrospective cohort study enrolled patients with CDH who underwent index cath from 2003 to 2022. Tricuspid annular plane systolic excursion z score, RV fractional area change, RV free wall and global longitudinal strain, left ventricular (LV) eccentricity index, RV/LV ratio, and pulmonary artery acceleration time were measured from preprocedure echocardiograms. Associations between hemodynamic values, echocardiographic measures, and survival were evaluated by Spearman correlation and Wilcoxon rank sum test, respectively.
Fifty-three patients (68% left-sided, 74% liver herniation, 57% extracorporeal membrane oxygenation, 93% survival) underwent cath (39 during index hospitalization, 14 later) including device closure of a patent ductus arteriosus in 5. Most patients (n = 31, 58%) were on pulmonary hypertension treatment at cath, most commonly sildenafil (n = 24, 45%) and/or intravenous treprostinil (n = 16, 30%). Overall, hemodynamics were consistent with precapillary pulmonary hypertension. Pulmonary capillary wedge pressure was >15 mm Hg in 2 patients (4%). Lower fractional area change and worse ventricular strain were associated with higher pulmonary artery pressure while higher LV eccentricity index and higher RV/LV ratio were associated with both higher pulmonary artery pressure and higher pulmonary vascular resistance. Hemodynamics did not differ based on survival status.
Worse RV dilation and dysfunction by echocardiogram correlate with higher pulmonary artery pressure and pulmonary vascular resistance on cath in this CDH cohort. These measures may represent novel, noninvasive clinical trial targets in this population.
To determine which patients with congenital diaphragmatic hernia (CDH) and pulmonary hypertension (PH) benefit from inhaled nitric oxide (iNO) treatment by comparing characteristics and outcomes of ...iNO responders to nonresponders.
We performed a retrospective chart review of infants with CDH treated at our center between 2011 and 2016. In a subset of patients, iNO was initiated for hypoxemia or echocardiographic evidence of extrapulmonary right to left shunting. Initial post-treatment blood gases were reviewed, and patients were classified as responders (increased PaO2 >20 mm Hg) or nonresponders. Baseline characteristics, echocardiograms and outcomes were compared between groups with Fisher exact tests and Mann-Whitney t tests, as appropriate.
During the study period, 95 of 131 patients with CDH (73%) were treated with iNO. All patients with pretreatment echocardiograms (n = 90) had echocardiographic evidence of PH. Thirty-eight (40%) patients met treatment response criteria. Responders had significant improvements in PaO2 (51 ± 3 vs 123 ± 7 mm Hg, P < .01), alveolar-arterial gradient (422 ± 30 vs 327 ± 27 mm Hg, P < .01), and PaO2 to FiO2 ratio (82 ± 10 vs 199 ± 15 mm Hg, P < .01). Nonresponders were more likely to have left ventricular systolic dysfunction (27% vs 8%, P = .03) on echocardiogram. Responders were less likely to require extracorporeal membrane support (50 vs 24%, P = .02).
iNO treatment is associated with improved oxygenation and reduced need for ECMO in a subpopulation of patients with CDH with PH and normal left ventricular systolic function.
Congenital diaphragmatic hernia (CDH) is a severe congenital anomaly which impairs normal pulmonary development leading to acute and chronic respiratory failure, pulmonary hypoplasia, pulmonary ...hypertension, and mortality. CDH is the most common non-cardiac indication for neonatal ECMO. Prenatal and postnatal predictors of CDH severity aid in patient selection. Centers vary in preferred mode of ECMO and timing of CDH repair. Survivors of severe CDH with ECMO are at risk for long-term sequelae including neurodevelopmental delays.
Congenital diaphragmatic hernia (CDH) is characterized by a developmental insult which compromises cardiopulmonary embryology and results in a diaphragmatic defect, allowing abdominal organs to ...herniate into the hemithorax. Among the significant pathophysiologic components of this condition is pulmonary hypertension (PH), alongside pulmonary hypoplasia and cardiac dysfunction. Fetal pulmonary vascular development coincides with lung development, with the pulmonary vasculature evolving alongside lung maturation. However, in CDH, this embryologic development is impaired which, in conjunction with external compression, stifle pulmonary vascular maturation, leading to reduced lung density, increased muscularization of the pulmonary vasculature, abnormal vascular responsiveness, and altered molecular signaling, all contributing to pulmonary arterial hypertension. Understanding CDH-associated PH (CDH-PH) is crucial for development of novel approaches and effective management due to its significant impact on morbidity and mortality. Antenatal and postnatal diagnostic methods aid in CDH risk stratification and, specifically, pulmonary hypertension, including fetal imaging and gas exchange assessments. Management strategies include lung protective ventilation, fluid optimization, pharmacotherapies including pulmonary vasodilators and hemodynamic support, and extracorporeal life support (ECLS) for refractory cases. Longitudinal re-evaluation is an important consideration due to the complexity and dynamic nature of CDH cardiopulmonary physiology. Emerging therapies such as fetal endoscopic tracheal occlusion and pharmacological interventions targeting key CDH pathophysiological mechanisms show promise but require further investigation. The complexity of CDH-PH underscores the importance of a multidisciplinary approach for optimal patient care and improved outcomes.
Pediatric extreme thrombocytosis (EXT, platelet count > 1000 x 10
3
/µL) is rare. In a single center retrospective analysis of hospitalized children with EXT, infants with congenital diaphragmatic ...hernia (CDH) were overrepresented. In general pediatric patients, EXT is usually secondary to infection or inflammation, but most of the 14 CDH patients with EXT had no identifiable inciting factor. Instead, there was evidence that splenic dysfunction and bone marrow hyperactivity underlied EXT in CDH patients. None were associated with bleeding or thrombosis. Our findings identify mechanisms underlying EXT, and aid clinical interpretation and management of EXT in the pediatric population.
Celotno besedilo
Dostopno za:
DOBA, IJS, IZUM, KILJ, NUK, PILJ, PNG, SAZU, UILJ, UKNU, UL, UM, UPUK
