Portal de Periódicos da CAPES

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

2014; Oxford University Press; Volume: 99; Issue: 11 Linguagem: Inglês

10.1210/jc.2014-1371

1945-7197

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

Oral and Maxillofacial Pathology

Fibrous dysplasia (FD) is a rare skeletal disorder, resulting in deformity, fracture, functional impairment, and pain. Bisphosphonates have been advocated as a potential treatment.To determine the efficacy of alendronate for treatment of FD.Two-year randomized, double-blind, placebo-controlled trial.Clinical research center.Forty subjects with polyostotic FD (24 adults, 16 children). Subjects were randomized and stratified by age.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.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.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.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.