[Clinical features of Chilean patients with Fibrous Dysplasia/McCune-Albright Syndrome]

Rev Med Chil. 2022 Oct;150(10):1275-1282. doi: 10.4067/S0034-98872022001001275.
[Article in Spanish]

Abstract

Background: Fibrous Dysplasia/McCune-Albright Syndrome (FD/MAS) is characterized by a spectrum of manifestations that may include fibrous dysplasia of bone and multiple endocrinopathies.

Aim: To describe the clinical spectrum, the study and follow-up of patients with FD/MAS cared at our institution.

Material and methods: Review of medical records of 12 pediatric and adult patients (11 women) who met the clinical and genetic diagnostic criteria for FD/ MAS.

Results: The patients' mean age at diagnosis was 4.9 ± 5.5 years. The most common initial clinical manifestation was peripheral precocious puberty (PPP) in 67% of patients and 75% had café-au-lait spots. Fibrous dysplasia was present in 75% of patients and the mean age at diagnosis was 7.9 ± 4.7 years. Ten patients had a bone scintigraphy, with an age at the first examination that varied between 2 and 38 years of age. The most frequent location of dysplasia was craniofacial and appendicular. No patient had a recorded history of cholestasis, hepatitis, or pancreatitis. In four patients, a genetic study was performed that was positive for the pathogenic variant of guanine nucleotide binding protein, alpha stimulating (GNAS).

Conclusions: These patients demonstrate the variable nature of the clinical presentation and study of FD/MAS. It is essential to increase the index of diagnostic suspicion and adherence to international recommendations.

Publication types

  • English Abstract

MeSH terms

  • Adolescent
  • Adult
  • Cafe-au-Lait Spots / genetics
  • Child
  • Child, Preschool
  • Chile / epidemiology
  • Female
  • Fibrous Dysplasia of Bone* / diagnostic imaging
  • Fibrous Dysplasia, Polyostotic* / diagnostic imaging
  • Fibrous Dysplasia, Polyostotic* / genetics
  • Humans
  • Puberty, Precocious* / etiology
  • Puberty, Precocious* / genetics
  • Young Adult