Pycnodysostosis with novel gene mutation and severe obstructive sleep apnoea: management of a complex case

BMJ Case Rep. 2013 Sep 20:2013:bcr2013200590. doi: 10.1136/bcr-2013-200590.

Abstract

Pycnodysostosis is a rare genetic disease. Impaired osteoclastic function is the basis for typical phenotypic features and bone fragility. The main differential diagnosis is osteopetrosis, also associated with altered bone remodelling, but with a more severe prognosis. We describe the case of an 8-year-old boy who presented life-threatening obstructive sleep apnoea successfully managed with non-invasive ventilation. Haematological overlap phenotype included anaemia and altered bone marrow, more common in osteopetrosis. Molecular analysis of the CTSK gene revealed a mutation not previously described in the literature.

Publication types

  • Case Reports

MeSH terms

  • Cathepsin K / genetics
  • Child
  • Glucosephosphate Dehydrogenase Deficiency / complications
  • Humans
  • Male
  • Mutation
  • Noninvasive Ventilation
  • Pycnodysostosis / complications
  • Pycnodysostosis / genetics
  • Pycnodysostosis / therapy*
  • Severity of Illness Index
  • Sleep Apnea, Obstructive / etiology
  • Sleep Apnea, Obstructive / therapy*

Substances

  • CTSK protein, human
  • Cathepsin K