A Randomized, Double Blind, Placebo-Controlled Trial of Alendronate Treatment for Fibrous Dysplasia of Bone

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-b...

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Published in:The journal of clinical endocrinology and metabolism 2014-11, Vol.99 (11), p.4133-4140
Main Authors: Boyce, Alison M, Kelly, Marilyn H, Brillante, Beth A, Kushner, Harvey, Wientroub, Shlomo, Riminucci, Mara, Bianco, Paolo, Robey, Pamela G, Collins, Michael T
Format: Article
Language:English
Subjects:
Adolescent
Adult
Alendronate - pharmacology
Alendronate - therapeutic use
Biological and medical sciences
Biomarkers - blood
Bone Density - drug effects
Bone Density Conservation Agents - pharmacology
Bone Density Conservation Agents - therapeutic use
Bone Remodeling - drug effects
Child
Collagen Type I - blood
Double-Blind Method
Endocrine Care
Endocrinopathies
Feeding. Feeding behavior
Female
Fibrous Dysplasia of Bone - blood
Fibrous Dysplasia of Bone - drug therapy
Fundamental and applied biological sciences. Psychology
Humans
Male
Medical sciences
Middle Aged
Osteocalcin - blood
Pain Measurement
Peptides - blood
Treatment Outcome
Vertebrates: anatomy and physiology, studies on body, several organs or systems
Vertebrates: endocrinology
Young Adult
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Summary: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.
ISSN:0021-972X
1945-7197
DOI:10.1210/jc.2014-1371