Wolf-Hirschhorn syndrome (WHS), a rare disorder determined by distal 4p deletion, is characterized by a pre and postnatal growth retardation, hypotonia, intellectual disability, epilepsy, craniofacial dysmorphism, and congenital fusion anomalies. The clinical aspects are dependent on the deletion' size. Our aim was to identify rare specific characteristics in a cohort of seven cases with 4p deletion and to assess the utility of Multiplex ligation-dependent probe amplification (MLPA) (cheap and sensitive test)-combined kits-as a diagnostic test and selection tool for cases that require other investigations (chromosomal microarray analysis-CMA, karyotype). For all cases we conducted a clinical examination with the main features identified: facial dysmorphism, intellectual disability, postnatal development delay, cardiac defects and hypotonia. In some cases, we observed seizures, structural brain abnormalities, immunodeficiencies, and renal anomalies. Prenatal growth retardation was detected in a relatively small number of cases, but postnatal growth failure was a constant feature. In all cases, the clinical diagnosis was confirmed by genetic analyses: karyotype and/or MLPA. In conclusion, renal and brain defects, as well as immunodeficiency are rare manifestations and should be looked for. Although CMA is the standard test, in our experience, MLPA is also a reliable screening method as the identified cases were either confirmed by MLPA or selected for further investigations.
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http://dx.doi.org/10.3390/children8090751 | DOI Listing |
Genet Med Open
March 2024
Department of Maternal-Fetal Biology, National Research Institute for Child Health and Development, Tokyo, Japan.
Purpose: Wolf-Hirschhorn syndrome (WHS), a contiguous gene syndrome caused by heterozygous deletions of the distal short arm of chromosome 4 that includes , reportedly causes specific DNA methylation signatures in peripheral blood cells. However, the genomic loci responsible for these signatures have not been elucidated. The present study aims to define the loci underlying WHS-related DNA methylation signatures and explore the role of in these signatures.
View Article and Find Full Text PDFLaryngoscope
November 2024
Department of Otolaryngology, Head and Neck Surgery, Mayo Clinic, Phoenix, Arizona, U.S.A.
Objectives: To assess the impact of body mass index (BMI) on hypoglossal nerve stimulator (HNS) implantation outcomes in pediatric patients with Down syndrome (DS). We compare outcomes of HNS implantation when comparing children based on overweight or obese status.
Methods: Retrospective cohort study of patients at a single tertiary pediatric hospital who underwent HNS implantation between 2022 and 2024.
Chromosome Res
November 2024
Department of Clinical Genomics, Mayo Clinic, 200 First Street SW, Rochester, MN, 55905, USA.
Chromosome 4p16.3 microdeletions are known to cause Wolf-Hirschhorn syndrome (WHS), which is characterized by a distinct craniofacial gestalt and multiple congenital malformations. The 4p16.
View Article and Find Full Text PDFCureus
September 2024
Anesthesiology, Lebanese American University Medical Center, Beirut, LBN.
Dev Med Child Neurol
October 2024
Department of Clinical Sciences, Orthopaedics, Lund University, Lund, Sweden.
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