A randomized, double blind, placebo-controlled trial of alendronate treatment for
fibrous dysplasia of bone.
Author(s): Boyce AM(1), Kelly MH, Brillante BA, Kushner H, Wientroub S, Riminucci M, Bianco
P, Robey PG, Collins MT.
Affiliation(s): Author information:
(1)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.
Publication date & source: 2014, J Clin Endocrinol Metab. , 99(11):4133-40
CONTEXT: 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.
DESIGN: Two-year randomized, double-blind, placebo-controlled trial.
SETTING: Clinical research center.
PATIENTS: Forty subjects with polyostotic FD (24 adults, 16 children). Subjects
were randomized and stratified by age.
INTERVENTIONS: 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.
MAIN OUTCOME MEASURES: 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.
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