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

Abstract

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.

• Authors: Alison M Boyce, Marilyn H Kelly, Beth A Brillante, Harvey Kushner, Shlomo Wientroub, Mara Riminucci, Paolo Bianco, Pamela G Robey, Michael T Collins
• Journal: The Journal of clinical endocrinology and metabolism (J Clin Endocrinol Metab) Vol. 99 Issue 11 Pg. 4133-40 (Nov 2014) ISSN: 1945-7197 [Electronic] United States
• PMID: 25033066 (Publication Type: Journal Article, Randomized Controlled Trial, Research Support, N.I.H., Intramural, Research Support, Non-U.S. Gov't)

Chemical References

• Biomarkers
• Bone Density Conservation Agents
• Collagen Type I
• Peptides
• collagen type I trimeric cross-linked peptide
• Osteocalcin
• Alendronate

Topics

• Adolescent
• Adult
• Alendronate (pharmacology, therapeutic use)
• Biomarkers (blood)
• Bone Density (drug effects)
• Bone Density Conservation Agents (pharmacology, therapeutic use)
• Bone Remodeling (drug effects)
• Child
• Collagen Type I (blood)
• Double-Blind Method
• Female
• Fibrous Dysplasia of Bone (blood, drug therapy)
• Humans
• Male
• Middle Aged
• Osteocalcin (blood)
• Pain Measurement
• Peptides (blood)
• Treatment Outcome
• Young Adult