Prognostic Factors From an Epidemiologic Evaluation of Fibrous Dysplasia of Bone in a Modern Cohort: The FRANCEDYS Study

Fibrous dysplasia of bone (FD) is a rare genetic but sporadic bone disease that can be responsible for bone pain, fracture, and bone deformity. The prognosis may be difficult to establish because of the wide spectrum of disease severity. We have analyzed the data from the French National Reference center for FD. We have established a database from standardized medical records. We have made descriptive statistics of the various forms of FD and examined the prognostic factors by multivariable logistic regression analysis, with a parsimonious stepwise method. The primary outcome was a clinically relevant composite index combining bone pain (visual analogic scale >3) and/or incident fracture. In our modern cohort of 372 patients, the median age at diagnosis was 23 years. The revealing symptom (at a median age of 18 years) was bone pain in 44% of patients and a fracture in 9%, but the diagnosis was fortuitous in 25% of cases. Monostotic forms represented 58% of patients and polyostotic forms 42%. The femur was the most commonly affected bone (44% of patients), followed by the skull (38%). Twelve percent of patients had McCune-Albright syndrome (MAS). With a median duration of follow-up of 7 years among 211 patients, we observed an incidence of fracture of 17% and 51% of patients had no bone pain at the end of follow-up (with or without bisphosphonate therapy). In univariate analysis, younger age at diagnosis, renal phosphate wasting, a polyostotic form, prevalent fracture, and bisphosphonate use were significant predictors. In the multivariate model, the polyostotic form and bisphosphonate use remained significant predictors. In conclusion, in a national referral center for FD, one patient on follow-up out of six had incident fracture. A polyostotic form was the main risk factor of a poorer outcome. (c) 2016 American Society for Bone and Mineral Research.