Literature DB >> 35916905

Bone Deformities and Kidney Failure: Coincidence of PHEX-Related Hypophosphatemic Rickets and m.3243A>G Mitochondrial Disease.

Simone Rask Nielsen1,2, Stinus Gadegaard Hansen3, Claus Bistrup4,5, Klaus Brusgaard6,5, Anja Lisbeth Frederiksen7,8.   

Abstract

X-linked hypophosphatemic rickets (XLH) and m.3243A>G mitochondrial disease share several clinical findings, including short stature, hearing impairment (HI), nephropathy, and hypertension. Here, we report on a case with the rare coincidence of these two genetic conditions. In early childhood, the patient presented with hypophosphatemia and bone deformities and was clinically diagnosed with XLH. This was genetically verified in adulthood with the identification of a de novo pathogenic deletion in phosphate-regulating endopeptidase homolog X-linked (PHEX). In addition, the patient developed HI and hypertension and when his mother was diagnosed with m.3243A>G, subsequent genetic testing confirmed the patient to carry the same variant. Over the next two decades, the patient developed progressive renal impairment however without nephrocalcinosis known to associate with XLH which could indicate an m.3243A>G-related kidney disease. Parallel with the progression of renal impairment, the patient developed hyperphosphatemia and secondary hyperparathyroidism. In conclusion, this case represents a complex clinical phenotype with the reversal of hypo- to hyperphosphatemia in XLH potentially mediated by the development of an m.3243A>G-associated nephropathy.
© 2022. The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature.

Entities:  

Keywords:  Bone deformities; Hypophosphatemic rickets; Kidney disease; PHEX; m.3243A>G

Year:  2022        PMID: 35916905     DOI: 10.1007/s00223-022-01010-x

Source DB:  PubMed          Journal:  Calcif Tissue Int        ISSN: 0171-967X            Impact factor:   4.000


  2 in total

Review 1.  Hearing impairment in familial X-linked hypophosphatemic rickets.

Authors:  Gadi Fishman; Denise Miller-Hansen; Cynthia Jacobsen; Virender K Singhal; Uri S Alon
Journal:  Eur J Pediatr       Date:  2004-10       Impact factor: 3.183

2.  MR imaging and proton MR spectroscopy in A-to-G substitution at nucleotide position 3243 of leucine transfer RNA.

Authors:  J Bowen; T Richards; K Maravilla
Journal:  AJNR Am J Neuroradiol       Date:  1998-02       Impact factor: 3.825

  2 in total

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