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A case report of multicentric carpotarsal osteolysis syndrome: Depiction of a debilitating disease course.

Jennifer Yee-Ming LiFanny Tsz-Wai HoBrian Hon-Yin ChungJoyce ChanBrian Hon-Yin ChungJoanna Yuet-Ling TungAlison Lap-Tak Ma
Published in: American journal of medical genetics. Part A (2024)
Multicentric carpotarsal osteolysis syndrome (MCTO) is a rare skeletal disorder characterized by progressive osteolysis involving the carpal and tarsal bones, and often associated with nephropathy. It is caused by heterozygous mutation in the MAF bZIP transcription factor B (MAFB) gene. Heterogeneous clinical manifestation and wide spectrum of disease severity have been observed in patients with MCTO. Here, we report a case of a male patient who presented with kidney failure in childhood with progressive disabling skeletal deformity. He was diagnosed with MCTO at 31-years-old, where a de novo pathogenic heterozygous variant in NM_005461.5:c.212C>A: p.(Pro71His) of the MAFB gene was identified. While there has been little data on the long-term prognosis and life expectancy of this disease, this case report sheds light on the debilitating disease course with multiple significant morbidities of a patient with MCTO throughout his lifetime of 33 years.
Keyphrases
  • case report
  • transcription factor
  • multiple sclerosis
  • early onset
  • copy number
  • genome wide
  • genome wide identification
  • electronic health record
  • big data
  • dna methylation
  • gene expression
  • deep learning