Bone turnover in children and adolescents with McCune-Albright syndrome treated with pamidronate for bone fibrous dysplasia

Calcif Tissue Int. 2002 Aug;71(2):121-8. doi: 10.1007/s00223-001-1098-7. Epub 2002 Jul 23.

Abstract

Bone fibrous dysplasia is one of the main features of McCune-Albright syndrome, a rare genetic condition caused by constitutive activating mutations of Gs-protein and defined by skin dysplasia, bone fibrous dysplasia, and autonomous multiple endocrinopathies. Raised serum alkaline phosphatase (ALP) and urinary hydroxyproline levels indicating bone metabolic hyperactivity have been reported in these patients. Encouraging therapeutic results have been achieved, mainly in adults, with pamidronate, an aminobisphosphonate. In this study we investigate newer bone metabolic indices in a cohort of 11 children and adolescents treated with pamidronate. Tenfold increases of bone ALP and urinary pyridinoline cross-links were found and osteocalcin levels were twofold higher compared with reference values. After treatment, significant decreases in bone ALP and cross-links (Wilcoxon test P < 0.06) were found. Bone mineral density (BMD) significantly increased during treatment. There were signs of radiological healing as thickening of the cortical bone was found in some cases.

Publication types

  • Clinical Trial

MeSH terms

  • Adolescent
  • Adult
  • Alkaline Phosphatase / blood
  • Anti-Inflammatory Agents / therapeutic use*
  • Bone Density / drug effects
  • Bone Resorption / drug therapy*
  • Bone and Bones / drug effects
  • Bone and Bones / metabolism*
  • Child
  • Collagen / urine
  • Collagen Type I
  • Diphosphonates / therapeutic use*
  • Female
  • Femur / diagnostic imaging
  • Femur / drug effects
  • Fibrous Dysplasia, Polyostotic* / drug therapy
  • Fibrous Dysplasia, Polyostotic* / metabolism
  • Fibrous Dysplasia, Polyostotic* / pathology
  • Humans
  • Lumbar Vertebrae / drug effects
  • Lumbar Vertebrae / metabolism
  • Male
  • Osteocalcin / blood
  • Pamidronate
  • Peptides / urine
  • Radiography
  • Treatment Outcome

Substances

  • Anti-Inflammatory Agents
  • Collagen Type I
  • Diphosphonates
  • Peptides
  • collagen type I trimeric cross-linked peptide
  • Osteocalcin
  • Collagen
  • Alkaline Phosphatase
  • Pamidronate