A randomized, double blind, placebo-controlled trial of alendronate treatment for fibrous dysplasia of bone

Alison M. Boyce, Marilyn H. Kelly, Beth A. Brillante, Harvey Kushner, Shlomo Wientroub, Mara Riminucci, Paolo Bianco, Pamela G. Robey, Michael T. Collins*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review


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.

Original languageEnglish
Pages (from-to)4133-4140
Number of pages8
JournalJournal of Clinical Endocrinology and Metabolism
Issue number11
StatePublished - 1 Nov 2014


Dive into the research topics of 'A randomized, double blind, placebo-controlled trial of alendronate treatment for fibrous dysplasia of bone'. Together they form a unique fingerprint.

Cite this