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Atypical, Composite, or Blended Phenotypes: How Different Molecular Mechanisms Could Associate in Double-Diagnosed Patients

Genes (Basel). 2022 Jul 19;13(7):1275. doi: 10.3390/genes13071275.

Abstract

In the last few years, trio-Whole Exome Sequencing (WES) analysis has revolutionized the diagnostic process for patients with rare genetic syndromes, demonstrating its potential even in non-specific clinical pictures and in atypical presentations of known diseases. Multiple disorders in a single patient have been estimated to occur in approximately 2-7.5% of diagnosed cases, with higher frequency in consanguineous families. Here, we report the clinical and molecular characterisation of eight illustrative patients for whom trio-WES allowed for identifing more than one genetic condition. Double homozygosity represented the causal mechanism in only half of them, whereas the other half showed peculiar multilocus combinations. The paper takes into consideration difficulties and learned lessons from our experience and therefore supports the powerful role of wide analyses for ascertaining multiple genetic diseases in complex patients, especially when a clinical suspicion could account for the majority of clinical signs. It finally makes clear how a patient's "deep phenotyping" might not be sufficient to suggest the presence of multiple genetic diagnoses but remains essential to validate an unexpected multilocus result from genetic tests.

Keywords: composite phenotype; deep phenotyping; double diagnosis; trio-WES.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Exome*
  • Family
  • Genetic Testing*
  • Homozygote
  • Phenotype

Grants and funding

The work was partially supported by the “PG23/FROM 2017 Call for Independent Research” as part of the RARE -Rapid Analysis for Rapid carE- project and by “Progetti di innovazione in ambito sanitario e socio sanitario Regione Lombardia, bando ex decreto n. 2713 del 28/02/2018” as part of the GENE—Genomic analysis Evaluation Network—project.