title: Effect of pamidronate treatment in children with polyostotic fibrous dysplasia of bone.
abstract: Intravenous infusions with the bisphosphonate compound pamidronate decrease bone pain and reportedly can lead to refilling of dysplastic lesions in adults with fibrous dysplasia (FD) of bone. Here we describe the effects of this treatment approach in 18 children and adolescents (age at start of therapy, 6.2-17.5 yr; eight girls) with polyostotic FD, who received pamidronate for 1.2-9.1 yr (median, 3.8 yr). Treatment cycles with pamidronate (1-1.5 mg/kg.d on 3 consecutive days) were given every 4 months. Levels of serum alkaline phosphatase and urinary collagen type I N-telopeptide were elevated at baseline and decreased continuously during the first 3 yr of therapy. There was no radiographic evidence of filling of lytic lesions or thickening of the bone cortex surrounding the lesions in any patient. Histomorphometric results in dysplastic bone tissue of patients receiving pamidronate (n = 7; time of therapy, 1.4-4.8 yr) were similar to those of patients without medical therapy (n = 9). No serious side effects were noted. In conclusion, pamidronate therapy appears to be safe in children and adolescents with polyostotic FD. However, we found no clear evidence that pamidronate has an effect on dysplastic lesions in such patients.
authors.affiliation: Genetics Unit, Shriners Hospital for Children, Québec, Canada H3G 1A6.
authors.forename: Horacio
authors.initials: H
authors.lastname: Plotkin
authors.forename: Frank
authors.initials: F
authors.lastname: Rauch
authors.forename: Leonid
authors.initials: L
authors.lastname: Zeitlin
authors.forename: Craig
authors.initials: C
authors.lastname: Munns
authors.forename: Rose
authors.initials: R
authors.lastname: Travers
authors.forename: Francis H
authors.initials: FH
authors.lastname: Glorieux
mesh_terms: D000293:Adolescent; D000469:Alkaline Phosphatase / Q000378:metabolism; D000893:Anti-Inflammatory Agents / Q000008:administration & dosage* / Q000009:adverse effects; D015415:Biomarkers; D001706:Biopsy; D001827:Body Height / Q000187:drug effects; D002118:Calcium / Q000652:urine; D002648:Child; D002675:Child, Preschool; D003094:Collagen / Q000652:urine; D024042:Collagen Type I; D003404:Creatinine / Q000652:urine; D004164:Diphosphonates / Q000008:administration & dosage* / Q000009:adverse effects; D005260:Female; D005269:Femur / Q000000981:diagnostic imaging / Q000254:growth & development / Q000378:metabolism; D005359:Fibrous Dysplasia, Polyostotic / Q000000981:diagnostic imaging / Q000188:drug therapy* / Q000378:metabolism; D005500:Follow-Up Studies; D006801:Humans; D007085:Ilium / Q000254:growth & development / Q000378:metabolism / Q000473:pathology; D008297:Male; D010146:Pain / Q000188:drug therapy; D000077268:Pamidronate; D010281:Parathyroid Hormone / Q000097:blood; D010455:Peptides / Q000652:urine; D011859:Radiography
chemical_list: D000893:Anti-Inflammatory Agents; D015415:Biomarkers; D024042:Collagen Type I; D004164:Diphosphonates; D010281:Parathyroid Hormone; D010455:Peptides; C052929:collagen type I trimeric cross-linked peptide; D003094:Collagen; D003404:Creatinine; D000469:Alkaline Phosphatase; D000077268:Pamidronate; D002118:Calcium