ESPE2024 Poster Category 2 Bone, Growth Plate and Mineral Metabolism (31 abstracts)
1Clinical Genetics Outpatient Clinic, Mother and Child Health Care Institute of Serbia "Dr Vukan Cupic", Belgrade, Serbia. 2Department of Endocrinology, Mother and Child Health Care Institute of Serbia "Dr Vukan Cupic", Belgrade, Serbia. 3Department of Orthopedic Surgery, Mother and Child Health Care Institute of Serbia "Dr Vukan Cupic", Belgrade, Serbia. 4Faculty of Medicine, University of Belgrade, Belgrade, Serbia
Background: Fibrous dysplasia (FD) is a rare bone disorder characterized by the proliferation of fibrous tissue within the bone, leading to pain, deformity, and an increased fracture risk. While management primarily focuses on symptomatic relief, surgical interventions, calcium supplementation, and vitamin D therapy, bisphosphonates have been used as a therapeutic option in children. However, studies of bisphosphonate efficiency have shown inconclusive results.
Objective: This retrospective analysis aimed to assess the efficacy and safety of bisphosphonate therapy in pediatric patients with the monostotic form of FD.
Methods: Medical records of five pediatric patients diagnosed with monostotic FD, who received bisphosphonate therapy between 2012 and 2024, were retrospectively analyzed. Data including demographics, clinical presentation, radiographic findings, biochemical markers, treatment regimens, and adverse events were assessed.
Results: The case series includes five patients (3 females, 2 males) with an age range at diagnosis of 10-13 years (median 12 years). The localization of FD was craniofacial in two patients and femoral in three. Stabilization of disease progression and alleviation of pain were achieved over the treatment course in all patients regardless of the administration route, while improvement in radiographic findings was verified in a subset of patients. Variability in adverse events, such as initial post-treatment hypocalcemia, gastrointestinal disturbances, and others, was observed.
Conclusion: Our study suggests that bisphosphonate therapy may offer benefits in the management of monostotic FD in pediatric patients, both during oral or intravenous regimens. Nevertheless, further prospective studies are warranted to elucidate long-term efficacy and safety profiles in pediatric patients with FD.
Keywords: Fibrous Dysplasia, Bisphosphonates, Pediatric.