O2153: Neurodevelopmental Outcomes in Children After Fetoscopic Endoluminal Tracheal Occlusion for Severe Congenital Diaphragmatic Hernia: Results from a Multidisciplinary Clinic

Background: Prior studies suggest that children with severe congenital diaphragmatic hernia (CDH) are at high risk for poorer neurodevelopmental outcomes. Fetoscopic endoluminal tracheal occlusion (FETO) is a prenatal therapy that improves lung function after birth. In this study, we compared early neurodevelopmental morbidity in young children with severe CDH who underwent FETO to those without fetal therapy.

Methods: We conducted a prospective study of severe CDH patients undergoing FETO at an experienced North American center from 2015-2021 (NCT02710968). Survivors were evaluated by an outpatient multidisciplinary team and compared to a prospective CDH cohort who underwent expectant fetal management. A post-hoc disease severity classification system was generated, based on observed/expected lung-to-head ratio, defect size, extracorporeal life support (ECLS) use, and liver herniation. Neurodevelopmental outcomes were assessed by the Capute Scales [Clinical Linguistic and Auditory Milestone Scales (CLAMS) and Cognitive Adaptive Test (CAT)] at 3, 12, and 24 months of age. These validated tests provide critical information regarding expressive and receptive language, and nonverbal, problem-solving skills in children < 3 years. A developmental quotient < 85 is indicative of language delay or visual motor and problem-solving on the CLAMS and CAT, respectively. Statistical analyses were performed with Wilcoxon Rank Sum, Chi-squared, and Fisher’s exact tests, as appropriate. Simple linear regression was utilized to compare trends in neurodevelopment scores (p≤0.05).

Results: Among 58 CDH patients, 8 (14%) were low severity, 13 (22%) were medium severity, 19 (33%) were high severity/non-FETO, and 18 (31%) were high severity/FETO. Survival was similar between FETO patients and high severity/non-FETO patients (67% vs. 63%, respectively; p=0.82). FETO was associated with significantly lower ECLS use compared to high severity/non-FETO patients (56% vs. 89%, p=0.02). At a median of 12.5 months, FETO was associated with language delay (CLAMS median DQ, 80.1 [67.6-86.7]), as opposed to low (93.5 [92-95]), medium (92 [87.6-93.3]), and high severity/non-FETO patients (90.5 [87.6-93.3]). However, at a median of 24.2 months, FETO scores improved, whereas high severity/non-FETO patients had decreased scores concerning for delay (median DQ (Difference in DQ over 2 years), 92.3 (+12.2) vs. 77.1 (-13.4), p=0.049) (Figure 1). On the initial CAT, FETO patients had visual motor and problem-solving delays, with a median DQ of 81.3 (62.1-89.4). However, FETO scores improved over 24 months at a similar rate to low severity patients (90.8 (+9.5) vs. 112.2 (+22.2), p=0.83), whereas medium (95.8 (-0.2), p=0.38) and high severity/non-FETO (87.5 (-3.0), p=0.28) had declining scores (Figure 2).

Conclusion: For the first time, our results suggest that fetal therapy with FETO is associated with improved neurodevelopmental outcomes at 24 months in patients with severe CDH. While long-term studies are needed, these data highlight the potential morbidity benefits of FETO and may inform prenatal counseling discussions with families.