title: A randomized, double blind, placebo-controlled trial of alendronate treatment for fibrous dysplasia of bone.
abstract: BACKGROUND
  Fibrous dysplasia (FD) is a rare skeletal disorder, resulting in deformity, fracture, functional impairment, and pain. Bisphosphonates have been advocated as a potential treatment.
  
  
  OBJECTIVE
  To determine the efficacy of alendronate for treatment of FD.
  
  
  METHODS
  Two-year randomized, double-blind, placebo-controlled trial.
  
  
  METHODS
  Clinical research center.
  
  
  METHODS
  Forty subjects with polyostotic FD (24 adults, 16 children). Subjects were randomized and stratified by age.
  
  
  METHODS
  Study drug was administered over a 24 month period in 6 month cycles (6 months on, 6 months off). Alendronate dosing was stratified: 40 mg daily for subjects >50 kg, 20 mg for 30-50 kg, 10 mg for 20-30 kg.
  
  
  METHODS
  Primary endpoints were bone turnover markers, including serum osteocalcin, and urinary NTX-telopeptides. Secondary endpoints included areal bone mineral density (aBMD), pain, skeletal disease burden score, and functional parameters including the 9-min walk test and manual muscle testing.
  
  
  RESULTS
  Clinical data was collected on 35 subjects who completed the study. There was a decline in NTX-telopeptides in the alendronate group (P = .006), but no significant difference in osteocalcin between groups. The alendronate group had an increase in areal BMD in normal bone at the lumbar spine (P = .006), and in predetermined regions of FD (P < .001). There were no significant differences in pain scores, skeletal disease burden scores, or functional parameters between the groups.
  
  
  CONCLUSIONS
  Alendronate treatment led to a reduction in the bone resorption marker NTX-telopeptides, and improvement in aBMD, but no significant effect on serum osteocalcin, pain, or functional parameters.
authors.affiliation: Skeletal Clinical Studies Unit, Craniofacial and Skeletal Diseases Branch (A.M.B., M.H.K., B.A.B., P.G.R., M.T.C.), National Institute of Dental and Craniofacial Research, National Institutes of Health, Bethesda, Maryland 20892; Division of Endocrinology and Diabetes (A.M.B.), Children's National Health System, Washington, DC 20010; Bone Health Program, Division of Orthopaedics and Sports Medicine (A.M.B.), Children's National Health System, Washington, DC 20010; BioMedical Computer Research Institute (H.K.), Philadelphia, Pennsylvania 19115; Department of Pediatric Orthopedics (S.W.), Dana Children's Hospital, Tel-Aviv Sourasky Medical Center, Sackler Faculty of Medicine, Tel-Aviv University, Israel 64239; Department of Molecular Medicine (M.R., P.B.), La Sapienza Universita di Roma, Rome, Italy 00185.
authors.forename: Alison M
authors.initials: AM
authors.lastname: Boyce
authors.forename: Marilyn H
authors.initials: MH
authors.lastname: Kelly
authors.forename: Beth A
authors.initials: BA
authors.lastname: Brillante
authors.forename: Harvey
authors.initials: H
authors.lastname: Kushner
authors.forename: Shlomo
authors.initials: S
authors.lastname: Wientroub
authors.forename: Mara
authors.initials: M
authors.lastname: Riminucci
authors.forename: Paolo
authors.initials: P
authors.lastname: Bianco
authors.forename: Pamela G
authors.initials: PG
authors.lastname: Robey
authors.forename: Michael T
authors.initials: MT
authors.lastname: Collins
mesh_terms: D000293:Adolescent; D000328:Adult; D019386:Alendronate / Q000494:pharmacology / Q000627:therapeutic use*; D015415:Biomarkers / Q000097:blood; D015519:Bone Density / Q000187:drug effects*; D050071:Bone Density Conservation Agents / Q000494:pharmacology / Q000627:therapeutic use*; D016723:Bone Remodeling / Q000187:drug effects*; D002648:Child; D024042:Collagen Type I / Q000097:blood; D004311:Double-Blind Method; D005260:Female; D005357:Fibrous Dysplasia of Bone / Q000097:blood / Q000188:drug therapy*; D006801:Humans; D008297:Male; D008875:Middle Aged; D015675:Osteocalcin / Q000097:blood; D010147:Pain Measurement; D010455:Peptides / Q000097:blood; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D015415:Biomarkers; D050071:Bone Density Conservation Agents; D024042:Collagen Type I; D010455:Peptides; C052929:collagen type I trimeric cross-linked peptide; D015675:Osteocalcin; D019386:Alendronate