Improved Management of Harlequin Ichthyosis With Advances in Neonatal Intensive Care

Pediatrics. 2017 Jan;139(1):e20161003. doi: 10.1542/peds.2016-1003. Epub 2016 Dec 20.

Abstract

Harlequin ichthyosis (HI) is the most severe phenotype of the autosomal recessive congenital ichthyoses. HI is caused by mutations in the lipid transporter adenosine triphosphate binding cassette A 12 (ABCA12). Neonates are born with a distinct clinical appearance, encased in a dense, platelike keratotic scale separated by deep erythematous fissures. Facial features are distorted by severe ectropion, eclabium, flattened nose, and rudimentary ears. Skin barrier function is markedly impaired, which can lead to hypernatremic dehydration, impaired thermoregulation, increased metabolic demands, and increased risk of respiratory dysfunction and infection. Historically, infants with HI did not survive beyond the neonatal period; however, recent advances in neonatal intensive care and coordinated multidisciplinary management have greatly improved survival. In this review, the authors combine the growing HI literature with their collective experiences to provide a comprehensive review of the management of neonates with HI.

Publication types

  • Review

MeSH terms

  • ATP-Binding Cassette Transporters / genetics
  • Combined Modality Therapy
  • DNA Mutational Analysis
  • Follow-Up Studies
  • Humans
  • Ichthyosis, Lamellar / diagnosis
  • Ichthyosis, Lamellar / genetics
  • Ichthyosis, Lamellar / mortality
  • Ichthyosis, Lamellar / therapy*
  • Infant
  • Infant, Newborn
  • Intensive Care, Neonatal / methods*
  • Interdisciplinary Communication
  • Intersectoral Collaboration
  • Phenotype
  • Prenatal Diagnosis
  • Prognosis
  • Survival Rate
  • Tertiary Care Centers

Substances

  • ABCA12 protein, human
  • ATP-Binding Cassette Transporters