Bisphosphonate therapy in pediatric fibrous dysplasia: A systematic review of clinical, biochemical, and radiological outcomes.

Background: Fibrous dysplasia (FD) is a rare bone disorder often presenting in children with bone pain, pathological fractures, and deformities. Bisphosphonates, as anti-resorptive agents, are increasingly used in pediatric FD to reduce symptoms and improve skeletal health. However, their efficacy and safety in this population remain incompletely defined. Methods: This systematic review was conducted according to PRISMA guidelines after registration on PROSPERO with ID: CRD420251102346. A comprehensive search of PubMed, Embase, Cochrane Library, and LILACS databases was performed up to June 25, 2025, using keywords ((FIBROUS DYSPLASIA) AND ((PAEDIATRIC) OR (CHILDREN)) AND (BISPHOSPHONATE)). Studies were included if they involved original clinical data, reported on bisphosphonate therapy in children (mean age <16 years), and were published in English. Data were extracted on demographics, clinical features, treatment regimen, biochemical outcomes, pain scores, complications, and radiological response. Results: A total of seven studies comprising 92 pediatric patients were included. Pamidronate was the most commonly used bisphosphonate, followed by zoledronic acid. All studies reported significant reduction in bone pain, and several noted decreased fracture frequency. Biochemical improvements, especially reductions in alkaline phosphatase, were consistently observed. However, radiological improvement and correction of skeletal deformities were limited. Bisphosphonate therapy was generally well-tolerated, with transient fever, mild infusion-related bone pain, and asymptomatic hypocalcemia being the most common adverse effects. Conclusion: Bisphosphonates are effective in improving clinical symptoms and biochemical markers in children with fibrous dysplasia but have limited impact on structural deformities. They demonstrate a favorable short-term safety profile.