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

Shelby R Sferra; Annalise B Penikis; Matthew Guo; Ahmet A Baschat; Peter J Mogayzel; Vera Joanna Burton; Shaun M Kunisaki

doi:10.1016/j.jpedsurg.2024.03.041

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

J Pediatr Surg. 2024 Jul;59(7):1271-1276. doi: 10.1016/j.jpedsurg.2024.03.041. Epub 2024 Mar 20.

Authors

Shelby R Sferra¹, Annalise B Penikis¹, Matthew Guo¹, Ahmet A Baschat², Peter J Mogayzel³, Vera Joanna Burton⁴, Shaun M Kunisaki⁵

Affiliations

¹ Division of General Pediatric Surgery, Department of Surgery, Johns Hopkins University School of Medicine, 1800 Orleans Street, Baltimore, MD 21287, USA.
² Johns Hopkins Center for Fetal Therapy, Department of Gynecology & Obstetrics, Johns Hopkins University School of Medicine, 600 N. Wolfe Street, Baltimore, MD 21287, USA.
³ Eudowood Division of Pediatric Pulmonary, Department of Pediatrics, Johns Hopkins University School of Medicine, 200 N. Wolfe Street, Baltimore, MD 21287, USA.
⁴ Neurology and Developmental Medicine, Department of Neurology, Johns Hopkins University School of Medicine, 801 N. Broadway Baltimore, MD 21205, USA.
⁵ Division of General Pediatric Surgery, Department of Surgery, Johns Hopkins University School of Medicine, 1800 Orleans Street, Baltimore, MD 21287, USA. Electronic address: skunisa1@jhmi.edu.

PMID: 38599907
DOI: 10.1016/j.jpedsurg.2024.03.041

Abstract

Background: 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 (n = 18) at a single North American center from 2015 to 2021 (NCT02710968). Outpatient survivors (n = 12) were evaluated by a multidisciplinary team and compared to expectantly managed CDH patients. Neurodevelopmental outcomes were assessed using the Capute Scales [Clinical Linguistic and Auditory Milestone Scales (CLAMS) and Cognitive Adaptive Test (CAT)], with a developmental quotient (DQ) < 85 indicative of at-risk for delay.

Results: At one year, 58% (n = 7) of FETO patients underwent evaluation, with notable concern for language delay (CLAMS median DQ, 80.1 [interquartile range, 67.6-86.7]). FETO scores improved by 24-months, whereas high severity/non-FETO scores declined [CLAMS median DQ (Difference in DQ), 92.3 (+12.2) vs. 77.1 (-13.4), respectively; p = 0.049]. On the initial CAT, FETO patients had concern for visual motor and problem-solving delays, with a median DQ of 81.3 (62.1-89.4). At 24-months, FETO patients had improving scores [Median CAT DQ, 90.8 (+9.5)], whereas high severity/non-FETO [87.5 (-3.0), p = 0.28] had declining scores.

Conclusion: These initial data suggest that FETO is associated with favorable neurodevelopmental outcomes at 24-months compared to severe CDH under expectant management.

Level of evidence: III.

Keywords: Congenital diaphragmatic hernia; Fetal therapy; Fetal tracheal occlusion; Outcomes.

MeSH terms

Child, Preschool
Developmental Disabilities / epidemiology
Developmental Disabilities / etiology
Female
Fetoscopy* / methods
Hernias, Diaphragmatic, Congenital* / surgery
Humans
Infant
Infant, Newborn
Male
Neurodevelopmental Disorders / epidemiology
Neurodevelopmental Disorders / etiology
Pregnancy
Prospective Studies
Trachea / surgery
Treatment Outcome